1 01:00:00:01 01:00:01:01

2 01:00:13:27 01:00:16:14
CAPTIONING MADE POSSIBLE BY
FRIENDS OF NCI

3 01:00:24:22 --:--:--:--
Woman: IN THE RECOVERY ROOM
AFTER THE DELIVERY, WE HAD THIS

4 01:00:29:05 --:--:--:--
BEAUTIFUL CHILD, AND WE WERE
KIND OF CONFUSED AS TO WHY SHE

5 01:00:32:23 --:--:--:--
WAS SO BLOND-HEADED.

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IT'S LIKE, WOW, YOU KNOW,
SHE LOOKS A LITTLE

7 01:00:36:23 --:--:--:--
LIKE AN ALBINO.

8 01:00:40:10 --:--:--:--
ASHLEY WAS A VERY LATE WALKER,
AND I NOTICED THAT SHE WAS

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BRUISING, AND I COULDN'T
FIGURE OUT WHERE THESE LITTLE

10 01:00:45:14 --:--:--:--
BRUISES WERE COMING FROM,
SO I BROUGHT HER TO

11 01:00:48:05 --:--:--:--
THE PEDIATRICIAN, AND I SAID,
"LOOK AT HER LITTLE SHINS.

12 01:00:50:17 --:--:--:--
THERE'S A BLEEDING PROBLEM."

13 01:00:52:03 --:--:--:--
HE SAID, "HER SKIN
IS FAIR, SO YOU SEE
BRUISES BETTER."

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AND I THOUGHT TO MYSELF,
"MY SKIN IS FAIR.

15 01:00:59:08 --:--:--:--
I DON'T SEE BRUISES.

16 01:01:00:19 01:01:03:10
I THINK THAT'S
A BUNCH OF MALARKEY."

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ONE NIGHT, 3:00 IN THE
MORNING, I HEARD WHIMPERING

18 01:01:09:00 --:--:--:--
IN ASHLEY'S ROOM, AND I WENT
IN THERE, AND HER CRIB WAS

19 01:01:13:18 --:--:--:--
FULL OF BLOOD.

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AND I SAW THAT SHE WAS
BLEEDING FROM HER RECTUM,

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AND I KNEW THAT WE WERE IN
TROUBLE, AND I KNEW THAT WE

22 01:01:22:19 01:01:24:23
HAD TO TAKE HER
TO THE HOSPITAL.

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SHE WAS IN THE INTENSIVE
CARE UNIT FOR WEEKS AND

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IN THE HOSPITAL FOR MONTHS.

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AND THAT WAS THE PUSH THAT
MADE ME REALLY START TO LOOK

26 01:01:36:28 --:--:--:--
INTO A LITTLE PAMPHLET I HAD
ON ALBINISM, AND THIS PAMPHLET

27 01:01:41:20 --:--:--:--
HAD THIS ONE LITTLE SENTENCE
OR TWO ABOUT AN EXTREMELY RARE

28 01:01:45:21 --:--:--:--
DISORDER CALLED
HERMANSKY-PUDLAK SYNDROME--

29 01:01:48:25 --:--:--:--
ALBINISM, BLEEDING PROBLEM,
VERY HORRIFIC LUNG DISEASE.

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WHEN ASHLEY WAS DIAGNOSED,
THERE WAS ABOUT 23 PEOPLE

31 01:01:58:04 --:--:--:--
IN THE U.S. MAINLAND THAT
WERE IDENTIFIED WITH HPS.

32 01:02:02:25 --:--:--:--
MY HUSBAND AND I FELT SO
ISOLATED AND SO FRIGHTENED,

33 01:02:08:23 --:--:--:--
AND I WANTED TO KNOW WHERE
THOSE 23 PEOPLE ARE, I WANT TO

34 01:02:12:09 --:--:--:--
KNOW WHAT THEY'RE DOING,
AND I WANT TO KNOW WHO'S

35 01:02:14:18 01:02:20:14
RESEARCHING THIS AND WHERE IS
THE CURE, AND I FOUND NOTHING.

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WHEN SOMEBODY TELLS YOU THAT
PEOPLE SUCCUMB IN THEIR 30s

37 01:02:35:07 --:--:--:--
AND 40s AND YOU HAVE THIS
YOUNG CHILD WITH YOU, YOU'RE

38 01:02:38:18 --:--:--:--
LIKE, "THAT'S A LONG TIME.

39 01:02:40:01 --:--:--:--
WE CAN CURE THIS DISEASE BY
THEN," AND YOU KIND OF FEEL

40 01:02:44:00 --:--:--:--
REALLY OK ABOUT THINGS.

41 01:02:46:00 --:--:--:--
I MEAN,
I CERTAINLY HAD, OBVIOUSLY,

42 01:02:49:00 --:--:--:--
SOME SENSE OF URGENCY,
BUT NOTHING LIKE THE URGENCY

43 01:02:53:00 01:02:55:06
I FEEL NOW.

44 01:03:06:15 --:--:--:--
ASHLEY'S LUNGS
ARE AFFECTED NOW.

45 01:03:10:06 --:--:--:--
THEY DON'T GIVE IT MORE THAN
10 YEARS, AND IT'S REALLY HARD

46 01:03:14:18 01:03:17:03
TO CURE A DISEASE IN
THAT LENGTH OF TIME.

47 01:03:29:09 --:--:--:--
SO YOU'RE EXPECTING
IT NOT TO AFFECT
HER LUNGS.

48 01:03:31:17 --:--:--:--
I STARTED THE HERMANSKY-PUDLAK
SYNDROME NETWORK BY TRYING TO

49 01:03:34:28 --:--:--:--
GATHER THE STORIES, ANYBODY
THAT I COULD FIND THAT HAD

50 01:03:39:23 --:--:--:--
A CHILD WITH HERMANSKY-PUDLAK
SYNDROME.

51 01:03:42:21 --:--:--:--
I STARTED WITH JUST, YOU KNOW,
ONE, TWO, THREE FAMILIES.

52 01:03:48:02 --:--:--:--
IT'S A REALLY AMAZING THING TO
SEE IT GO FROM 23 TO NUMBERS

53 01:03:53:27 --:--:--:--
THAT I CAN'T HANDLE.

54 01:03:55:22 01:03:58:18
[MUSIC PLAYING]

55 01:04:09:22 01:04:11:13
[INDISTINCT] BABY!

56 01:04:21:21 01:04:24:07
[CHEERING AND LAUGHING]

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CAN I HAVE SOMEBODY
TELL ME HOW THEY PUT
EYELINER ON?

58 01:04:29:02 --:--:--:--
I USE THE LIQUID.

59 01:04:30:03 --:--:--:--
YOU USE THE LIQUID?

60 01:04:31:04 --:--:--:--
YEAH.

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I USE THE PENCIL, BUT I
USUALLY USE IT INSIDE THE EYE.

62 01:04:35:09 --:--:--:--
WHAT DO YOU GUYS DO
ABOUT DARK CIRCLES?

63 01:04:37:20 --:--:--:--
I KNOW WE HAVE LIGHTER
SKIN, SO IT SHOWS.

64 01:04:40:13 --:--:--:--
UNDER YOUR EYES, YOU
CAN SEE YOUR VEINS.

65 01:04:42:11 --:--:--:--
SOMETIMES IT'S REALLY HARD
TO FIND EYE SHADOW THAT WILL

66 01:04:44:24 --:--:--:--
REALLY MATCH THE SKIN.

67 01:04:46:25 --:--:--:--
THIS IS WHAT I LIKE.

68 01:04:47:25 --:--:--:--
IT'S, LIKE,
FOR YOUR EYEBROWS.

69 01:04:49:06 --:--:--:--
IT HAS A LITTLE BRUSH.

70 01:04:52:01 --:--:--:--
I USE THAT, TOO.

71 01:04:53:01 --:--:--:--
THAT'S REALLY
GOOD, TOO.

72 01:04:54:14 01:04:58:13
IT'S THE WHEAT COLOR, AND IT
MATCHES THE BLOND HAIR GREAT.

73 01:05:04:21 --:--:--:--
HOW EXCITING IT IS THAT
OUR GROUP HAS GROWN.

74 01:05:08:19 --:--:--:--
JUST REMEMBER THAT THE
FIRST TWO CONFERENCES WERE

75 01:05:11:16 --:--:--:--
IN MY HOME.

76 01:05:13:13 --:--:--:--
ONE YEAR, PEOPLE HAD TO HAVE
THEIR BLOOD DRAWN DOWNSTAIRS,

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AND THE OTHER YEAR, THEY
HAD TO PEE IN BUCKETS.

78 01:05:19:08 --:--:--:--
[LAUGHTER]

79 01:05:21:03 --:--:--:--
AREN'T YOU GLAD YOU
MISSED THAT ONE?

80 01:05:23:09 --:--:--:--
AND I COOKED, WHICH WAS
THE SCARIEST PART.

81 01:05:26:11 --:--:--:--
Donna, voice-over:
PEOPLE SAY, HERMANSKY-PUDLAK
SYNDROME IS SO RARE, AND I'M

82 01:05:30:14 --:--:--:--
LIKE, "RARE? I'M HAVING
AN EPIDEMIC HERE."

83 01:05:32:24 --:--:--:--
GOOD MORNING, EVERYBODY.

84 01:05:33:25 --:--:--:--
MY NAME IS NOEL
ESTRADA HERNANDEZ.

85 01:05:35:13 --:--:--:--
I'M ORIGINALLY
FROM PUERTO RICO.

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I LIVE IN A PLACE CALLED
READING, WHICH IS VERY CLOSE

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TO LONDON, IN ENGLAND.

88 01:05:41:12 01:05:43:25
[SPEAKING FOREIGN
LANGUAGE]

89 01:05:47:17 --:--:--:--
WE LOVE TO BE HERE.

90 01:05:48:28 --:--:--:--
WE DIDN'T EXPECT SO MANY
PEOPLE TO HAVE HPS, SO IT'S

91 01:05:52:09 --:--:--:--
GREAT TO BE HERE.

92 01:05:53:20 --:--:--:--
THIS IS MY FIRST
YEAR HERE.

93 01:05:55:21 --:--:--:--
I'M REALLY HAPPY
TO BE HERE.

94 01:05:57:12 --:--:--:--
I, UM...

95 01:06:00:29 --:--:--:--
I DIDN'T KNOW
THE SEVERITY OF HPS
UNTIL I GOT

96 01:06:07:18 --:--:--:--
HERE THIS MORNING.

97 01:06:09:07 --:--:--:--
Donna: YOU GUYS DON'T
UNDERSTAND HOW MUCH
YOU HELP RESEARCH BY JUST

98 01:06:12:25 --:--:--:--
COMING TO THE CONFERENCE,

99 01:06:15:02 --:--:--:--
BECAUSE DOCTORS ARE HERE.

100 01:06:16:22 --:--:--:--
THEY GET TO TALK TO PEOPLE.

101 01:06:18:12 --:--:--:--
THAT'S AIDEN.

102 01:06:19:21 01:06:21:21
[APPLAUSE]

103 01:06:24:25 --:--:--:--
SO EVERY SINGLE ONE OF YOU IS
WORKING TO HELP HIS FUTURE.

104 01:06:29:07 01:06:31:23
[APPLAUSE]

105 01:06:52:02 --:--:--:--
WHEN ASHLEY WAS DIAGNOSED,
ONE OF THE DOCTORS THAT I WAS

106 01:06:55:08 --:--:--:--
SPEAKING TO MENTIONED THAT
HPS IS KIND OF LIKE AN NIH

107 01:06:58:12 --:--:--:--
DISEASE, AND I NEVER
HAD HEARD OF THE NIH.

108 01:07:01:06 --:--:--:--
I REALLY DIDN'T
KNOW WHAT IT WAS.

109 01:07:04:05 --:--:--:--
I CALLED UP, AND
DR. GAHL GOT ON THE PHONE.

110 01:07:09:00 --:--:--:--
[GASP]

111 01:07:10:20 --:--:--:--
I GOT A DOCTOR AT THE NIH.

112 01:07:12:29 --:--:--:--
I WAS LIKE, "OH, MY GOD,
I'VE REALLY OUTDONE
MYSELF TODAY."

113 01:07:16:18 --:--:--:--
BUT I WAS JUST CRYING.
I WAS LIKE--

114 01:07:19:29 --:--:--:--
I WAS SO BLUBBERY,
I'M SURPRISED HE EVEN

115 01:07:23:25 --:--:--:--
CONTINUED THE CONVERSATION,
BUT I SAID NOT ONLY DO I HAVE

116 01:07:28:20 --:--:--:--
THIS DAUGHTER WITH HPS,
BUT I HAVE THESE PEOPLE,

117 01:07:31:09 --:--:--:--
AND I HAVE THESE CASES, AND WE
NEED RESEARCH, AND WE DON'T

118 01:07:34:17 01:07:37:15
HAVE RESEARCH, AND COULD
YOU PLEASE HELP US?

119 01:07:40:18 --:--:--:--
Man, voice-over:
ADVOCACY GROUPS FOR RARE
DISEASES START OUT WITH ONE

120 01:07:44:15 --:--:--:--
STRIKE AGAINST THEM.

121 01:07:45:15 --:--:--:--
GOOD MORNING.

122 01:07:46:15 --:--:--:--
NICE TO SEE YOU.

123 01:07:47:23 --:--:--:--
NAMELY, THAT THERE
ARE VERY FEW OF THEM.

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THEY DON'T HAVE MUCH LOBBYING
EFFORT, AND OTHER PEOPLE DON'T

125 01:07:54:14 --:--:--:--
UNDERSTAND WHY ONE SHOULD
STUDY A RARE DISORDER INSTEAD

126 01:07:57:09 --:--:--:--
OF A COMMON DISORDER.

127 01:07:58:17 --:--:--:--
IF YOU NEED TO ASK
A QUESTION,
NOW YOU CAN ASK.

128 01:08:01:20 --:--:--:--
Gahl, voice-over:
DONNA APPELL USED HER
PERSUASIVE POWERS

129 01:08:05:22 --:--:--:--
TO INFLUENCE US
TO STUDY HPS,

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SO SHE WOULD SEND ME
NOTES AND SORT OF KEEP

131 01:08:11:24 --:--:--:--
IN TOUCH AND JUST BE VERY,
VERY NICE, BUT I COULD NOT

132 01:08:16:13 --:--:--:--
COMMIT SIGNIFICANT AMOUNTS
OF GOVERNMENT RESOURCES,

133 01:08:19:11 --:--:--:--
OR ESPECIALLY MY OWN TIME,
TO A DISORDER THAT I THOUGHT

134 01:08:22:15 --:--:--:--
WAS NOT GOING TO MAKE PROGRESS
IN TERMS OF OUR UNDERSTANDING

135 01:08:29:02 01:08:31:13
OF THE CELL BIOLOGY
OR THE BIOCHEMISTRY.

136 01:08:33:10 --:--:--:--
HPS IS AN EXAMPLE OF A VERY
RARE DISEASE THAT MIGHT REVEAL

137 01:08:37:18 --:--:--:--
SOME THINGS THAT ARE HELPFUL
FOR COMMON DISEASES OR

138 01:08:40:24 --:--:--:--
FOR THE REGULAR COMMUNITY.

139 01:08:42:27 --:--:--:--
SOMETIMES WE THINK OF A DEFECT
IN A SINGLE GENE AS HAVING

140 01:08:46:18 --:--:--:--
A SINGLE EFFECT, BUT
HPS IS INTERESTING TO US

141 01:08:50:10 --:--:--:--
BECAUSE HERE, A SINGLE GENE
ABNORMALITY AFFECTS SEVERAL

142 01:08:54:14 --:--:--:--
DIFFERENT PARTS OF THE BODY.

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IN THE SKIN, IT CAUSES A
LACK OF PIGMENTATION, WHICH

144 01:08:59:27 --:--:--:--
PRODUCES ALBINISM.

145 01:09:01:26 --:--:--:--
IN THE BLOOD, THE PLATELETS
AREN'T FORMED PROPERLY,

146 01:09:04:29 --:--:--:--
CAUSING A BLEEDING DISORDER.

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AND IN THE LUNGS, IT
CAUSES FIBROSIS, A SCARRING

148 01:09:10:08 --:--:--:--
OF THE LUNG TISSUE WHICH
IS ULTIMATELY FATAL.

149 01:09:13:22 --:--:--:--
HPS CAN HELP US TO UNDERSTAND
THESE PROCESSES, SO ALTHOUGH

150 01:09:18:11 --:--:--:--
HPS AFFECTS A SMALL NUMBER OF
PEOPLE, WE ARE REALLY HOPING

151 01:09:22:21 --:--:--:--
THAT STUDYING IT WILL
EVENTUALLY LEAD TO THERAPIES

152 01:09:25:17 01:09:27:21
FOR MORE COMMON DISEASES.

153 01:09:30:19 --:--:--:--
WOW.

154 01:09:31:19 --:--:--:--
THIS IS WHEN WE
FIRST--WE HAVE PICTURES.

155 01:09:33:28 --:--:--:--
LOOK HOW CUTE.

156 01:09:35:19 --:--:--:--
Gahl, voice-over: WHEN
ASHLEY FIRST CAME TO US
AT AGE 3, WE THOUGHT WE HAD 2

157 01:09:39:27 --:--:--:--
TO 3 DECADES IN WHICH TO
DEVELOP A THERAPY FOR HER.

158 01:09:44:08 --:--:--:--
WE NO LONGER FEEL THAT WAY.

159 01:09:45:28 --:--:--:--
SEE HOW THIS THING
SPINS.

160 01:09:47:19 --:--:--:--
Gahl, voice-over:
WE'VE MADE PROGRESS
OVER THE LAST DECADE.

161 01:09:50:20 --:--:--:--
WE HAVE A POTENTIAL DRUG.

162 01:09:52:22 --:--:--:--
WE HAVE A TEAM OF INDIVIDUALS
WORKING ON THIS, INSTEAD

163 01:09:55:23 --:--:--:--
OF JUST ONE OR TWO.

164 01:09:57:29 --:--:--:--
STILL, OVERALL, WE'RE MORE
NERVOUS ABOUT THIS, AND HER

165 01:10:02:00 --:--:--:--
CASE IN PARTICULAR, SO WE'RE
WORKING--I WOULD SAY WE'RE

166 01:10:06:04 --:--:--:--
REDOUBLING OUR EFFORTS.

167 01:10:07:25 --:--:--:--
PLEASE HELP ME IN
WELCOMING DR. WILLIAM GAHL.

168 01:10:10:19 --:--:--:--
[APPLAUSE]

169 01:10:13:06 --:--:--:--
Gahl, voice-over:
PATIENTS WITH HPS PLACE
A LOT OF THEIR HOPE

170 01:10:16:22 --:--:--:--
IN A MEDICAL THERAPY.

171 01:10:18:24 --:--:--:--
RIGHT NOW, THE ONLY MEDICAL
THERAPY THAT'S REALLY VIABLE

172 01:10:22:12 --:--:--:--
IS THIS PIRFENIDONE STUDY.

173 01:10:24:07 --:--:--:--
Gahl, onscreen: BUT
IT'S CRITICAL THAT WE RECEIVE
ENROLLMENT OF 22 MORE PATIENTS

174 01:10:29:05 --:--:--:--
TO GET UP TO OUR 39 BECAUSE
THE SOONER WE ENROLL THE

175 01:10:32:12 --:--:--:--
PATIENTS, THE SOONER WE CAN
DO AN INTERIM ANALYSIS TO

176 01:10:35:18 --:--:--:--
DEMONSTRATE IF THIS WORKS.

177 01:10:38:04 --:--:--:--
MY NAME IS ISRAEL RODRIGUEZ.

178 01:10:39:15 --:--:--:--
MOST PEOPLE CALL ME IZZY.

179 01:10:41:26 --:--:--:--
WE NEED TO MAKE RECRUITMENT
FOR THIS PARTICULAR PROTOCOL

180 01:10:45:14 --:--:--:--
OUR TOP PRIORITY, AND I FEEL
LIKE I CAN SAY THAT AS SOMEONE

181 01:10:49:04 --:--:--:--
WHO'S NOT A SCIENTIST AND
WHO'S JUST LEARNING

182 01:10:51:15 --:--:--:--
AND LIVING WITH HPS.

183 01:10:53:06 --:--:--:--
Donna, voice-over:
TO DO OUTREACH AND FIND THE
CANDIDATES FOR THE DRUG TRIAL

184 01:10:58:20 --:--:--:--
IS AN ARDUOUS TASK BECAUSE
YOU CAN RECRUIT QUITE A LARGE

185 01:11:03:26 --:--:--:--
NUMBER OF PEOPLE, BUT PEOPLE
MAY BE DISQUALIFIED BECAUSE

186 01:11:08:19 --:--:--:--
OF ONE THING OR ANOTHER.

187 01:11:10:06 --:--:--:--
PLUS, WE ARE NOT A
LARGE NUMBER OF PEOPLE.

188 01:11:13:09 --:--:--:--
Izzy: DR. GAHL SAID WE
NEED 22 PATIENTS.

189 01:11:15:25 --:--:--:--
I THINK WE EACH NEED TO
MAKE THIS OUR PERSONAL

190 01:11:18:18 --:--:--:--
RESPONSIBILITY TO HELP
FIND QUALIFIED PEOPLE.

191 01:11:23:05 --:--:--:--
USUALLY IT TAKES--
FOR A RARE DISEASE LIKE
THIS, I WOULD SAY

192 01:11:27:26 --:--:--:--
IT WOULD BE ONE TO TWO YEARS,
IF THE FDA ACCEPTS THE DATA,

193 01:11:33:04 --:--:--:--
BEFORE IT WOULD BE APPROVED.

194 01:11:34:29 --:--:--:--
SO WE'RE TALKING ABOUT AT
LEAST IN THE 5-YEAR RANGE

195 01:11:38:29 --:--:--:--
FOR APPROVAL OF PIRFENIDONE
FOR HERMANSKY-PUDLAK SYNDROME.

196 01:11:44:01 --:--:--:--
DEEP BREATH IN.

197 01:11:45:29 --:--:--:--
BLOW OUT.

198 01:11:47:08 --:--:--:--
NICE, STEADY FLOW.

199 01:11:50:01 --:--:--:--
KEEP PUSHING.

200 01:11:51:13 01:11:53:09
KEEP PUSHING.

201 01:11:55:10 --:--:--:--
Woman, voice-over:
IT'S KIND OF FUNNY BECAUSE
I GO BACK AND I LOOK AT MY

202 01:11:57:03 --:--:--:--
MEDICAL RECORDS, AND THEY
DESCRIBED HPS TO A "T,"

203 01:12:01:05 --:--:--:--
BUT NOBODY KNEW WHAT IT WAS.

204 01:12:03:01 --:--:--:--
OK, HEATHER.
COME OFF NOW, PLEASE.

205 01:12:05:21 01:12:08:19
[COUGHING]

206 01:12:09:23 --:--:--:--
Heather, voice-over:
I WENT TO A NEW DOCTOR

207 01:12:12:15 --:--:--:--
AND FILLED OUT
THE MEDICAL HISTORY

208 01:12:14:04 --:--:--:--
FORM, AND THE DOCTOR STARTED
ASKING ME QUESTIONS ABOUT HPS

209 01:12:17:15 01:12:19:29
THAT I DIDN'T KNOW
THE ANSWER TO.

210 01:12:21:20 --:--:--:--
SO I WENT BACK TO MY OFFICE
AND I GOOGLED IT AND ABOUT HAD

211 01:12:24:15 --:--:--:--
A HEART ATTACK RIGHT
THERE IN THE OFFICE.

212 01:12:27:01 --:--:--:--
IN THE FIRST PARAGRAPH,
IT WOULD SAY, YOU KNOW,

213 01:12:29:08 --:--:--:--
AT THE END OF IT, "USUALLY
FATAL IN THE THIRD TO FOURTH

214 01:12:31:24 --:--:--:--
DECADE," AND I WAS 29.

215 01:12:34:19 --:--:--:--
I'M LIKE, "HELLO."

216 01:12:37:11 --:--:--:--
MUCH OF YOUR LUNG LOOKS
NORMAL, BUT THEN THERE ARE

217 01:12:40:06 --:--:--:--
SOME AREAS WHERE WE CAN
SEE SOME GROUND GLASS.

218 01:12:42:29 --:--:--:--
I DON'T KNOW IF YOU CAN SEE
HERE, BUT THERE'S A DIFFERENCE

219 01:12:45:14 --:--:--:--
IN TERMS OF THE LEVEL
OF DARKNESS OF THE GRAY.

220 01:12:48:24 --:--:--:--
Heather, voice-over:
I'M IN SUCH AN EARLY STAGE
OF PULMONARY FIBROSIS.

221 01:12:51:27 --:--:--:--
IF YOU COULD TIME GOING INTO A
PROTOCOL PERFECTLY, I TIMED IT

222 01:12:55:16 --:--:--:--
JUST RIGHT, BECAUSE I
WAS REALLY AT THE TOP END

223 01:12:58:29 --:--:--:--
OF ELIGIBILITY, WHICH I THINK
IS THE BEST PLACE TO BE,

224 01:13:04:02 --:--:--:--
BECAUSE IT ALLOWS ME SOME ROOM
TO FALL IF THE TRIAL DRAGS

225 01:13:09:13 --:--:--:--
ON FOR A WHILE, AND WE DON'T
KNOW WHO'S GETTING A PLACEBO

226 01:13:12:11 01:13:14:00
AND WHO'S NOT.

227 01:13:16:29 --:--:--:--
ON THE ONE HAND, YOU'RE
HAPPY TO GET INTO THE TRIAL,

228 01:13:19:28 --:--:--:--
BUT ON THE OTHER HAND,
IT'S KIND OF A JARRING WAKE-UP

229 01:13:22:16 --:--:--:--
CALL BECAUSE YOU REALIZE
THAT YOU WOULDN'T BE GETTING

230 01:13:25:11 --:--:--:--
ADMITTED TO THE TRIAL
IF YOU WEREN'T SICK.

231 01:13:28:23 --:--:--:--
HERE'S WHAT THEY LOOK LIKE.

232 01:13:32:24 --:--:--:--
I REMEMBER ONE DAY LOOKING
AT THE PILLS IN MY HAND

233 01:13:34:16 --:--:--:--
AND GOING, "YOU KNOW, I
WONDER IF THE TERM SUGAR PILL

234 01:13:36:25 --:--:--:--
"IS LITERAL.

235 01:13:38:02 --:--:--:--
"LIKE, IF I CUT THESE OPEN
AND TASTE THEM, LIKE, WHAT

236 01:13:41:10 --:--:--:--
WOULD IT BE?"

237 01:13:42:22 --:--:--:--
I KNEW I'D GET IN TROUBLE FOR
THAT, SO I DID NOT DO THAT,

238 01:13:46:05 --:--:--:--
BUT YOU WONDER, AND THEN EVERY
LITTLE THING THAT GOES WRONG,

239 01:13:49:20 --:--:--:--
YOU'RE LIKE, "OH, GOSH,
MY STOMACH IS UPSET.

240 01:13:52:11 --:--:--:--
"GREAT! MAYBE THAT'S GOOD,
BECAUSE THAT MEANS I'M GETTING

241 01:13:55:14 --:--:--:--
THE STUFF."

242 01:13:56:27 --:--:--:--
OR IF YOU GET A RASH,
IT'S LIKE, "RASH. YES!"
YOU KNOW?

243 01:14:01:10 --:--:--:--
GOOD.
NOW FOLLOW MY FINGER
ALL THE WAY IN.

244 01:14:03:07 --:--:--:--
FOLLOW IT RIGHT TO
THE TIP OF YOUR NOSE.

245 01:14:05:24 --:--:--:--
EXCELLENT. PERFECT.

246 01:14:07:21 --:--:--:--
Heather, voice-over:
I DON'T WANT TO SOUND LIKE
I'M COMPLAINING, BECAUSE I'M

247 01:14:09:00 --:--:--:--
REALLY GRATEFUL TO BE IN
THE TRIAL, BUT THERE ARE

248 01:14:12:03 --:--:--:--
SOME DRAWBACKS.

249 01:14:13:18 --:--:--:--
THE BIGGEST ONE FOR ME
IS THE TIME INVOLVED.

250 01:14:16:07 --:--:--:--
IT'S 3 WEEKS OUT OF THE YEAR,
SO IT PRETTY MUCH EATS UP MY

251 01:14:19:25 --:--:--:--
VACATION TIME.

252 01:14:21:27 --:--:--:--
EVERY TIME THERE'S A HOLIDAY,
EVERY MEMBER OF MY FAMILY IS--

253 01:14:25:07 --:--:--:--
BECAUSE THEY LOVE ME; IT'S
GREAT--THEY'RE LIKE, "PLEASE

254 01:14:29:03 --:--:--:--
"COME TO OUR HOUSE
FOR THANKSGIVING.

255 01:14:30:27 --:--:--:--
COME TO OUR HOUSE
FOR CHRISTMAS."

256 01:14:32:28 --:--:--:--
I CAN'T GO ANYWHERE BECAUSE
MY TIME IS HERE, AND SOMETIMES

257 01:14:38:20 --:--:--:--
THAT CAN BE, I THINK, HARD
FOR PEOPLE TO UNDERSTAND.

258 01:14:42:02 --:--:--:--
IT'S NOT THAT YOU
DON'T WANT TO SEE THEM.

259 01:14:44:07 01:14:48:05
IT'S THAT THIS IS
JUST SO IMPORTANT
THAT IT HAS TO COME FIRST.

260 01:14:50:14 --:--:--:--
VIBRATION.

261 01:14:52:08 --:--:--:--
VIBRATION.

262 01:14:54:00 --:--:--:--
Heather, voice-over: BUT
ONE OF THE THINGS THAT YOU
CANNOT DO ON THIS PARTICULAR

263 01:14:56:04 --:--:--:--
DRUG TRIAL IS YOU
CAN'T GET PREGNANT.

264 01:14:59:06 --:--:--:--
AND I'M NOT MARRIED RIGHT NOW.

265 01:15:01:23 --:--:--:--
IT'S NOT LIKE I'M ITCHING
TO GET PREGNANT TOMORROW,

266 01:15:04:04 --:--:--:--
BUT I AM 34.

267 01:15:05:14 --:--:--:--
THIS IS PROBABLY GOING TO
DRAG ON FOR AT LEAST 5 YEARS.

268 01:15:09:03 --:--:--:--
IT SEEMED SILLY BECAUSE IT
WASN'T LIKE I WAS GIVING UP

269 01:15:11:27 --:--:--:--
SOMETHING THAT WAS GOING TO
BE IMMEDIATE, BUT IT WAS KIND

270 01:15:17:11 --:--:--:--
OF EMOTIONAL FOR ME,
AND I DON'T THINK ANYBODY

271 01:15:20:28 --:--:--:--
REALIZED THAT.

272 01:15:22:14 01:15:27:06
IT WAS JUST ONE OF THE THINGS
ON THE LIST, YOU KNOW, SO...

273 01:15:30:12 --:--:--:--
THE SITUATION SUCKS.

274 01:15:32:02 --:--:--:--
I MEAN, LET'S FACE IT.

275 01:15:34:07 --:--:--:--
YOU WOULD NEVER WANT TO BE IN
THIS POSITION, BUT IF YOU HAVE

276 01:15:36:29 --:--:--:--
TO BE IN IT, IT IS GRATIFYING
TO KNOW THAT YOU COULD PLAY

277 01:15:41:02 --:--:--:--
A ROLE IN HELPING TO FIND
A CURE, HELPING TO FIND

278 01:15:43:19 --:--:--:--
A TREATMENT, SO THAT THE NEXT
GENERATION OF PEOPLE WITH HPS

279 01:15:46:22 01:15:49:24
DON'T HAVE TO FACE SOME OF THE
PROBLEMS THAT WE'RE FACING.

280 01:15:56:19 --:--:--:--
[COMPUTER BEEPS]

281 01:15:59:08 --:--:--:--
HOLA.

282 01:16:00:08 --:--:--:--
HOLA.

283 01:16:02:10 --:--:--:--
DO YOU SEE ME?

284 01:16:03:28 --:--:--:--
YES.

285 01:16:05:29 --:--:--:--
OOH! I LIKE
THAT COLOR SHIRT.

286 01:16:07:20 --:--:--:--
THAT'S REALLY
PRETTY.

287 01:16:09:29 --:--:--:--
OH. THANK YOU.

288 01:16:11:21 --:--:--:--
ARE YOU EXCITED
ABOUT NEXT WEEK?

289 01:16:13:14 --:--:--:--
YEAH, I'M REALLY
EXCITED.

290 01:16:16:20 --:--:--:--
I CAN'T WAIT.

291 01:16:17:24 --:--:--:--
AND I CAN'T WAIT UNTIL
WE SEE EACH OTHER.

292 01:16:20:12 --:--:--:--
I'M EXCITED.

293 01:16:22:25 --:--:--:--
Donna, voice-over: WE
MADE THIS APPOINTMENT TO
GO AND DO THIS LECTURE

294 01:16:26:02 --:--:--:--
AT THE DIVISION OF THE BLIND
SERVICES IN THE ORLANDO AREA,

295 01:16:30:18 --:--:--:--
AND MY DAUGHTER WAS WITH ME,
AND SITTING IN THE CORNER WAS

296 01:16:33:25 --:--:--:--
THIS YOUNG MAN, AND
HE WAS RATHER CUTE.

297 01:16:36:25 --:--:--:--
Mervin: I GOT TO
MAKE SURE THAT
MY TESTS DON'T CONFLICT.

298 01:16:39:29 --:--:--:--
Donna, voice-over: EVEN
THOUGH THEY'RE LEGALLY
BLIND, THEY SOMEHOW KIND

299 01:16:42:28 --:--:--:--
OF SAW EACH OTHER, AND THE
TWO OF THEM STARTED TO TALK,

300 01:16:46:08 --:--:--:--
AND SOMEBODY INVITED SOMEBODY
TO GO TO THE MOVIES THAT

301 01:16:49:05 --:--:--:--
NIGHT, AND THEIR RELATIONSHIP
KIND OF BLOSSOMED FROM THERE.

302 01:16:52:26 --:--:--:--
Mervin: NOT AS SWEET
AS YOU, THOUGH.

303 01:16:54:12 --:--:--:--
OH...ACTUALLY, I'M GOING
IN FOR REMICADE TONIGHT.

304 01:16:58:15 --:--:--:--
BUT I DON'T KNOW.

305 01:16:59:29 --:--:--:--
I'M TRYING TO DEBATE
WHETHER TO BRING MY
PSYCHOLOGY WITH ME

306 01:17:04:13 --:--:--:--
TO MY REMICADE AND
STUDY WHILE I'M IN BED.

307 01:17:08:13 --:--:--:--
Ashley, voice-over:
DATING SOMEBODY WITH HPS,
THEY KIND OF KNOW A BIT

308 01:17:13:08 --:--:--:--
OF YOUR LIFE AND WHAT
YOU REALLY GO THROUGH

309 01:17:15:00 --:--:--:--
IN YOUR LIFE,
YOU KNOW?

310 01:17:16:22 --:--:--:--
AND HE REALLY UNDERSTANDS ME,
AND I THINK IT'S VERY COOL TO

311 01:17:22:04 --:--:--:--
HAVE SOMEBODY THAT YOU CAN
TALK TO ABOUT VERY SIMILAR

312 01:17:27:00 --:--:--:--
SITUATIONS THAT YOU'RE HAVING.

313 01:17:29:20 01:17:31:11
HERE YOU GO, SWEETIE.

314 01:17:32:29 --:--:--:--
YEAH. THAT'LL WORK
JUST FINE.

315 01:17:35:25 --:--:--:--
Recording: FOR A PRESCRIPTION
REFILL, PLEASE ENTER

316 01:17:37:27 --:--:--:--
A TELEPHONE NUMBER--

317 01:17:39:08 --:--:--:--
[BEEPING]

318 01:17:42:01 --:--:--:--
PLEASE ENTER YOUR
PRESCRIPTION NUMBER.

319 01:17:43:18 --:--:--:--
[BEEPING]

320 01:17:46:25 --:--:--:--
Donna, voice-over: SO
WE'RE REALLY LUCKY TO HAVE
ASHLEY'S LITTLE NURSING

321 01:17:49:17 --:--:--:--
STATION OVER HERE.

322 01:17:51:14 --:--:--:--
I TRY TO KEEP HER
MEDICATIONS PRETTY ORGANIZED.

323 01:17:53:27 --:--:--:--
I ACTUALLY KEEP HER NIGHTTIME
MEDICINES TOWARD THIS END

324 01:17:57:10 --:--:--:--
AND HER DAYTIME MEDICINES.

325 01:17:59:10 --:--:--:--
SHE HAS INHALERS.

326 01:18:01:11 --:--:--:--
IF SHE GETS HURT,
I HAVE DDAVP.

327 01:18:04:12 --:--:--:--
I HAVE THIS READY TO GO,
SO IF SHE'S IN THE HOUSE

328 01:18:07:23 --:--:--:--
AND BLEEDS FROM FALLING.

329 01:18:10:01 --:--:--:--
SHE HAS TO HAVE AN EPIPEN ON
STANDBY BECAUSE HER THROAT

330 01:18:13:18 --:--:--:--
CLOSES WITH I.V. DDAVP.

331 01:18:15:27 --:--:--:--
WE HAVE HER EMERGENCY
MEDICATIONS, AND I HAVE TO

332 01:18:18:18 --:--:--:--
ROTATE THE MEDICATIONS
BECAUSE THIS WILL GET EXPIRED.

333 01:18:21:08 --:--:--:--
WE TEST ASHLEY'S STOOL.

334 01:18:23:03 --:--:--:--
SHE BLEEDS A LOT.

335 01:18:24:08 --:--:--:--
WE NEED TO BE ABLE TO TEST
FOR HER BLOOD SUGAR.

336 01:18:28:12 01:18:31:11
YOU KNOW, YOU GOT TO
DO WHAT YOU GO TO DO.

337 01:18:38:14 --:--:--:--
IT'S DEFINITELY ONE THING
TO TAKE CARE OF AN EPISODIC

338 01:18:43:04 --:--:--:--
ILLNESS AND RISE TO THAT
OCCASION AND TAKE CARE OF THAT

339 01:18:47:02 --:--:--:--
PERSON, AND THEN THEY GET
WELL, AND THEN YOU HAVE POST-

340 01:18:51:05 --:--:--:--
TRAUMATIC STRESS SYNDROME,
WHERE YOU CAN FALL

341 01:18:53:13 --:--:--:--
APART AND GO,
"OH, MY GOD, THAT WAS QUITE

342 01:18:56:22 --:--:--:--
A LIFE-ALTERING EXPERIENCE
FOR MOI," AND MOVE ON,

343 01:19:01:24 --:--:--:--
AND DEALING WITH CHRONIC
ILLNESS, IT'S NOT LIKE THAT.

344 01:19:05:21 --:--:--:--
IT'S LIKE RUNNING A
MARATHON THAT NEVER ENDS.

345 01:19:08:04 --:--:--:--
WHAT OCCURRED THAT CAUSED
YOU TO FIND THE DIAGNOSIS?

346 01:19:12:06 --:--:--:--
LIKE, WHAT WAS THAT THING THAT
HAPPENED IN YOUR LIFE WITH

347 01:19:15:20 --:--:--:--
THE CHILDREN THAT MADE YOU
GO AND SEARCH?

348 01:19:20:04 --:--:--:--
WHEN ELENA WAS BORN, YOU
CHECK THEIR EYES RIGHT AWAY.

349 01:19:23:11 --:--:--:--
SHE WASN'T EVEN OUT
ALL THE WAY,

350 01:19:26:01 --:--:--:--
AND IT WAS LIKE,
"HOW DO HER EYES LOOK?"

351 01:19:27:29 --:--:--:--
OF COURSE, IT DOESN'T SHOW UP
RIGHT AWAY, BUT I REMEMBER

352 01:19:30:24 --:--:--:--
THE DAY I WAS SITTING IN THE
CHAIR ROCKING HER, AND ALL

353 01:19:33:05 --:--:--:--
OF A SUDDEN, IT STARTED.

354 01:19:35:08 --:--:--:--
I HAD PRAYED THE WHOLE TIME
I WAS PREGNANT THAT IF THE

355 01:19:38:08 --:--:--:--
SECOND ONE HAD IT, THAT GOD
HAD A PLAN, AND THE THAT TWO

356 01:19:41:04 --:--:--:--
TOGETHER WOULD DO SOMETHING,
AND SO...YOU KNOW, I DON'T

357 01:19:45:27 --:--:--:--
HAVE THE WISDOM OF THE PLAN
YET, BUT I FIGURE, THEY'RE

358 01:19:49:18 --:--:--:--
THERE FOR EACH OTHER, AND
I CAN SEE THAT UNFOLDING.

359 01:19:53:02 --:--:--:--
THEY'RE NOT ALONE.

360 01:19:54:13 --:--:--:--
THEY DO HAVE EACH OTHER,
AND I'M THANKFUL FOR THAT--

361 01:19:57:04 --:--:--:--
ALONG WITH EVERYBODY
ELSE'S KIDS.

362 01:20:00:20 --:--:--:--
BUT, YEAH, THERE ARE
MOMENTS WHERE YOU SIT
THERE AND WONDER,

363 01:20:06:24 --:--:--:--
WHY HAS THIS HAPPENED,
OR YOUR PART ABOUT THAT,

364 01:20:09:17 --:--:--:--
AND EVERYTHING LIKE
THAT.

365 01:20:10:24 --:--:--:--
I GUESS THE THING
THAT I USED TO
OFFSET THAT WAS,

366 01:20:17:05 --:--:--:--
I GAVE YOU THIS DISEASE,
BUT, UH...

367 01:20:21:23 01:20:23:19
[CRYING]

368 01:20:28:22 --:--:--:--
THE GOOD LORD
GAVE YOU A FATHER
WHO WILL TEACH YOU HOW

369 01:20:33:02 --:--:--:--
TO LIVE WITH IT.

370 01:20:37:17 01:20:39:25
AND THAT OFFSETS
IT, TOO.

371 01:20:43:16 --:--:--:--
[SPEAKING SPANISH]

372 01:20:46:09 01:20:48:19
HOW WOULD OUR
LIVES BE LIKE...

373 01:20:51:17 --:--:--:--
[SPEAKING SPANISH]

374 01:20:53:08 --:--:--:--
IF WE DIDN'T
HAVE A CHILD...

375 01:20:55:08 --:--:--:--
[SPEAKING SPANISH]

376 01:20:56:20 --:--:--:--
WITH THAT CONDITION?

377 01:20:58:17 01:21:00:14
[SPEAKING SPANISH]

378 01:21:05:13 --:--:--:--
BECAUSE PERHAPS IT'S THOSE
GUILT FEELINGS OR THOSE OTHER

379 01:21:09:11 --:--:--:--
FEELINGS THAT WE FEEL ARE
WHAT BRINGS US HERE, WHAT

380 01:21:12:24 --:--:--:--
BROUGHT US HERE.

381 01:21:13:29 --:--:--:--
[SPEAKING SPANISH]

382 01:21:18:19 --:--:--:--
AND IT'S WHAT MOTIVATES US TO
FIGHT FOR THEM AND TO FIGHT

383 01:21:21:15 --:--:--:--
FOR ALL THE OTHERS
WHO ALSO HAVE THIS.

384 01:21:24:26 --:--:--:--
THE QUESTION IS, WHO
WOULD WE BE IF THEY
WERE NOT CHILDREN

385 01:21:29:15 --:--:--:--
WITH ALBINISM OR
CHILDREN WITH HPS?

386 01:21:32:11 --:--:--:--
A LITTLE CALMER.

387 01:21:33:19 01:21:34:21
[LAUGHTER]

388 01:21:39:29 --:--:--:--
WANT ME TO TAKE YOUR CANE?

389 01:21:41:14 --:--:--:--
GOOD MORNING, ASHLEY.

390 01:21:42:15 --:--:--:--
HOW ARE YOU?

391 01:21:43:17 --:--:--:--
I'M GOOD.
HOW ARE YOU?

392 01:21:46:07 --:--:--:--
Gahl: WE WOULD NOT BE STUDYING
HERMANSKY-PUDLAK SYNDROME IF

393 01:21:48:11 --:--:--:--
IT WEREN'T FOR DONNA, BECAUSE
WE NEED PATIENTS, AND SHE'S

394 01:21:53:14 --:--:--:--
BEEN ABLE TO HELP US TO
RECRUIT PATIENTS TO

395 01:21:56:15 --:--:--:--
THE PROTOCOLS THAT ALLOW US
TO STUDY THIS DISEASE HERE

396 01:21:59:09 --:--:--:--
AT THE NATIONAL
INSTITUTES OF HEALTH.

397 01:22:00:20 --:--:--:--
WE'RE JUST MAKING SURE
YOUR VALVE IS NORMAL.

398 01:22:05:00 --:--:--:--
Gahl, voice-over: STILL,
WE'RE UNCOMFORTABLE OVER
ASHLEY'S LUNG SITUATION,

399 01:22:11:04 01:22:13:24
TO BE PERFECTLY BLUNT.

400 01:22:17:00 --:--:--:--
HER STEROID USE EXCLUDED
HER FROM THE CURRENT

401 01:22:20:09 --:--:--:--
PIRFENIDONE TRIAL.

402 01:22:23:12 --:--:--:--
FOR A GROUP OF INDIVIDUALS
FOR WHOM THIS TRIAL OFFERS ONE

403 01:22:29:17 01:22:34:20
OF THE ONLY HOPES, THIS WAS
A HEARTBREAKING DISCUSSION.

404 01:22:37:00 --:--:--:--
Donna, voice-over:
I FELT SAD THAT MY DAUGHTER
WAS EXCLUDED FROM THE PROTOCOL

405 01:22:41:00 --:--:--:--
BECAUSE SHE'S ON REMICADE
AND NEEDS REMICADE.

406 01:22:44:16 --:--:--:--
I AM GOING TO RECRUIT PEOPLE
OTHER THAN MY DAUGHTER TO GET

407 01:22:49:20 --:--:--:--
THIS DRUG THAT I THINK WOULD
BE VERY HELPFUL FOR HER.

408 01:22:54:20 --:--:--:--
THESE ARE YOUR RECORDS,
YOUR NUMBERS HERE, AND SOME

409 01:22:59:10 --:--:--:--
OF THIS STUFF WE GET FOR OUR
CLINICAL RESEARCH PURPOSES,

410 01:23:04:01 --:--:--:--
AND SOME OF THESE THINGS
ARE IMPORTANT FOR YOUR
MEDICAL CARE.

411 01:23:06:17 --:--:--:--
Gahl, voice-over: BECAUSE
THE OTHER OPTION IS,
ESSENTIALLY, TO GET A LUNG

412 01:23:10:07 --:--:--:--
TRANSPLANT, BUT THERE HAVE
BEEN ONLY 2 LUNG TRANSPLANTS

413 01:23:13:00 --:--:--:--
DONE ON HERMANSKY-PUDLAK
SYNDROME ANYWHERE

414 01:23:15:27 --:--:--:--
IN THE WORLD, AT
LEAST THAT I KNOW OF,

415 01:23:18:17 --:--:--:--
AND WHAT THAT TELLS YOU IS
THAT IT'S VERY DIFFICULT TO

416 01:23:21:13 --:--:--:--
GET A LUNG TRANSPLANT
FOR AN HPS PATIENT.

417 01:23:24:11 --:--:--:--
WHAT WE REALLY WANT TO
FOLLOW IS THE LUNG ISSUES.

418 01:23:29:02 --:--:--:--
Donna, voice-over:
I'VE LEARNED WHAT
GOOD SCIENCE IS NOW,

419 01:23:31:18 --:--:--:--
AND I FEEL THAT IT'S ALSO THE
RIGHT THING TO DO, BUT I'M HER

420 01:23:36:08 --:--:--:--
MOM, AND EVERYTHING I'VE
WORKED FOR AND WORKED TOWARDS

421 01:23:40:19 --:--:--:--
IS TO TRY TO GET A CURE OR
TREATMENT FOR HERMANSKY-PUDLAK

422 01:23:44:07 --:--:--:--
SYNDROME, SO TO EXCLUDE HER
WAS A LITTLE DIFFICULT,

423 01:23:49:10 --:--:--:--
YOU KNOW, BUT IT'S THE RIGHT
THING TO DO, AND IT'S WHAT WE

424 01:23:53:12 --:--:--:--
HAVE TO DO.

425 01:23:55:28 --:--:--:--
AND THAT'S A REAL GOOD
BASELINE TO HAVE, TOO.

426 01:23:59:05 --:--:--:--
Gahl, voice-over:
THERE ARE CONFLICTS INVOLVED
IN CLINICAL RESEARCH

427 01:24:01:26 --:--:--:--
ON THE PART OF THE
PHYSICIAN INVESTIGATOR,

428 01:24:04:14 --:--:--:--
BECAUSE A PERSONAL
RELATIONSHIP ESTABLISHES THAT

429 01:24:08:04 --:--:--:--
REALLY TRANSCENDS THE
SCIENTIFIC AND THE FIXED

430 01:24:13:05 --:--:--:--
CLINICAL PARAMETERS.

431 01:24:15:00 --:--:--:--
IT REALLY IS AN INTERPERSONAL
RELATIONSHIP, AND THERE'S

432 01:24:17:27 --:--:--:--
A CERTAIN AMOUNT OF
EMOTIONALITY INVOLVED IN THAT.

433 01:24:21:25 --:--:--:--
SO YOU WANT YOUR PATIENT TO
BE ENROLLED IN A PROTOCOL,

434 01:24:27:03 --:--:--:--
AND YOU WOULDN'T BE DOING THE
PROTOCOL UNLESS YOU BELIEVED

435 01:24:29:14 --:--:--:--
THERE WERE A REASONABLE
CHANCE THAT IT'S BENEFICIAL,

436 01:24:33:07 --:--:--:--
SO THERE'S DISAPPOINTMENT WHEN
A PATIENT IS NOT ELIGIBLE,

437 01:24:42:16 --:--:--:--
AND THERE'S A TEMPTATION TO
SKIRT THE RULES OR TO FUDGE

438 01:24:50:00 --:--:--:--
THINGS A TAD, AND ONE HAS TO
BE DILIGENT NOT TO DO THAT

439 01:24:54:26 01:24:57:28
BECAUSE IT RUINS THE STUDIES.

440 01:25:00:03 --:--:--:--
BUT WE'RE ALL HUMAN BEINGS,
AND THOSE THINGS ACTUALLY

441 01:25:03:13 01:25:05:09
DO ENTER IN.

442 01:25:08:27 --:--:--:--
SO, HEATHER, WE HAVE THESE
RESULTS, PULMONARY FUNCTION

443 01:25:13:19 --:--:--:--
TESTS, WHICH IS REALLY SORT
OF THE CRITICAL ISSUE HERE.

444 01:25:18:00 --:--:--:--
IF WE WERE TO ENTER YOU
INTO THE STUDY NOW--

445 01:25:19:15 --:--:--:--
YOU COULDN'T.

446 01:25:20:25 --:--:--:--
WE COULDN'T ENTER YOU INTO
THE STUDY BECAUSE YOU'RE

447 01:25:22:23 --:--:--:--
DOING TOO WELL.

448 01:25:23:23 --:--:--:--
HAVE YOU BEEN
EXERCISING OR SOMETHING?

449 01:25:26:25 --:--:--:--
YES, BUT LIKE
MORE THAN USUAL.

450 01:25:30:06 --:--:--:--
I DON'T OWN A CAR, AND I
LIVE IN KANSAS, SO I

451 01:25:32:11 --:--:--:--
WALK EVERYWHERE.

452 01:25:33:18 --:--:--:--
WELL, THIS ISN'T
KANSAS ANYMORE.

453 01:25:35:11 --:--:--:--
RIGHT.

454 01:25:36:12 --:--:--:--
HA HA HA!

455 01:25:37:26 --:--:--:--
BUT YOU'RE DOING EXTREMELY
WELL, AND WE'RE REALLY PLEASED

456 01:25:39:15 --:--:--:--
WITH THAT.

457 01:25:40:15 --:--:--:--
GREAT, GREAT.

458 01:25:42:07 --:--:--:--
ONCE AGAIN, THIS DOESN'T
REALLY DEMONSTRATE THAT,
FIRST OF ALL, YOU'RE

459 01:25:46:22 --:--:--:--
ON PIRFENIDONE OR THAT THE
PIRFENIDONE IS HELPING.

460 01:25:50:04 --:--:--:--
WE ONLY CAN DETERMINE THAT
FROM THE SERIES OF PATIENTS

461 01:25:53:23 --:--:--:--
THAT WE SEE, WHEN THE
STATISTICIANS PUT IT

462 01:25:56:02 --:--:--:--
ALL TOGETHER.

463 01:25:57:18 --:--:--:--
BUT FOR YOU PERSONALLY,
THIS IS REALLY GREAT NEWS.

464 01:26:00:13 --:--:--:--
YES.

465 01:26:02:00 --:--:--:--
SO WE'LL SEE YOU
AGAIN IN 4 MONTHS?

466 01:26:03:00 --:--:--:--
YEAH.

467 01:26:04:03 --:--:--:--
OK. GREAT. THANKS
FOR BEING PART OF THIS.

468 01:26:05:18 01:26:06:22
THANK YOU.

469 01:26:10:00 --:--:--:--
GOOD TO SEE YOU.

470 01:26:11:02 --:--:--:--
HOW ARE YOU?

471 01:26:12:17 01:26:14:18
WE'RE ALL READY FOR YOU.

472 01:26:17:28 --:--:--:--
Donna, voice-over:
IT'S EXCITING TO BE HERE.

473 01:26:19:05 --:--:--:--
IT'S EXCITING TO TRY TO
MAKE A DIFFERENCE.

474 01:26:21:21 --:--:--:--
CMS WAS SUPPOSED
TO AND PROMISED TO
GET A GENETIC...

475 01:26:26:12 --:--:--:--
Donna, voice-over:
WHEN I'M HERE, I KIND OF
THINK ABOUT THE BIG PICTURE

476 01:26:29:05 --:--:--:--
AND WHAT'S AT STAKE,
AND CERTAINLY WHAT'S AT STAKE

477 01:26:32:05 --:--:--:--
IS MY DAUGHTER'S WELFARE,
AND EVERYBODY WITH GENETIC

478 01:26:35:06 --:--:--:--
DISORDERS' WELFARE, SO THERE'S
TOO MUCH TO LOSE NOT TRYING TO

479 01:26:39:00 --:--:--:--
DO SOMETHING.

480 01:26:40:21 --:--:--:--
IT'S A LITTLE HARD TO DO IT
AND A LITTLE LONELY TO DO IT

481 01:26:42:21 --:--:--:--
BY YOURSELF, SO IT'S
REALLY GREAT TO BE PART

482 01:26:45:00 --:--:--:--
OF THE GENETIC ALLIANCE
BECAUSE YOU ALL HAVE

483 01:26:47:06 --:--:--:--
A GENETIC PROBLEM.

484 01:26:48:14 --:--:--:--
NO MATTER WHAT THE HECK
IT'S CALLED, WE ALL HAVE

485 01:26:50:01 --:--:--:--
THE SAME ISSUES.

486 01:26:51:11 --:--:--:--
I'M WITH THE
TRIMETHYLAMINURIA
FOUNDATION.

487 01:26:53:28 --:--:--:--
IT'S A METABOLIC
DISORDER.

488 01:26:56:10 --:--:--:--
Man: I'M REPRESENTING
THE 200,000-PLUS
INDIVIDUALS

489 01:27:00:07 --:--:--:--
WITH MARFAN SYNDROME
OR RELATED CONNECTIVE

490 01:27:01:20 --:--:--:--
TISSUE DISORDERS.

491 01:27:02:29 --:--:--:--
THERE IS A GENETIC TEST,
BUT IF PEOPLE DO NOT KNOW THAT

492 01:27:05:23 --:--:--:--
THEY NEED TO START LOOKING AT
WHAT SOME OF THESE SYMPTOMS

493 01:27:08:09 --:--:--:--
MEAN, THEY WOULDN'T KNOW TO
ACTUALLY ASK FOR THAT TEST.

494 01:27:11:15 --:--:--:--
WE'RE REALLY THRILLED TO HAVE
YOU COME HERE TODAY FROM ALL

495 01:27:14:08 --:--:--:--
OVER THE COUNTRY TO JOIN
US ON SEVERAL ISSUES.

496 01:27:18:04 --:--:--:--
Same woman, voice-over:
GENETIC ALLIANCE
IS A COALITION

497 01:27:19:20 --:--:--:--
OF ABOUT 650 DISEASE
ADVOCACY GROUPS.

498 01:27:22:10 --:--:--:--
WE'RE COVERING ALL 100
SENATE OFFICES TODAY.

499 01:27:24:25 --:--:--:--
THAT'S PRETTY PHENOMENAL.

500 01:27:26:17 --:--:--:--
Same woman, voice-over:
AND I BELIEVE REALLY ALL THESE
DISEASES ARE CONNECTED

501 01:27:28:21 --:--:--:--
AND THAT THE GENETICS
COMMUNITY, THE RARE DISEASE

502 01:27:30:20 --:--:--:--
COMMUNITY, REALLY IS ALL
PART OF A LARGE "WE."

503 01:27:33:16 --:--:--:--
WE ARE IN THIS BOAT TOGETHER.

504 01:27:35:17 --:--:--:--
BUT TELL ME ABOUT THE TRIAL.

505 01:27:36:22 --:--:--:--
WHAT'S HAPPENING WITH THAT?

506 01:27:38:24 --:--:--:--
WE ARE STILL RECRUITING,
SO WE'RE WORKING HARD

507 01:27:41:28 --:--:--:--
AT TRYING TO GET
PEOPLE INTO IT STILL,
WHICH IS PRETTY

508 01:27:46:22 --:--:--:--
DISAPPOINTING BECAUSE
THE PEOPLE THAT WENT

509 01:27:48:09 --:--:--:--
IN IN THE VERY
BEGINNING WERE EXPECTING
THIS 2-3 YEAR FINISH

510 01:27:52:21 --:--:--:--
DATE, BUT IF WE'RE NOT
LOADED YET...
RIGHT.

511 01:27:56:05 --:--:--:--
SO IT'S VERY
HEARTBREAKING.

512 01:27:57:09 --:--:--:--
SO HOW MANY
PEOPLE DO YOU NEED?

513 01:27:58:14 --:--:--:--
WE WOULD BE HAPPY
WITH 40.

514 01:28:00:03 --:--:--:--
WE'RE AT ABOUT 24.

515 01:28:01:22 --:--:--:--
SO WE'RE DOING
ANOTHER CONFERENCE
IN PUERTO RICO.

516 01:28:04:05 --:--:--:--
OH, OK.

517 01:28:05:09 --:--:--:--
THAT'S A LOGISTIC
NIGHTMARE.

518 01:28:07:07 --:--:--:--
WE'RE KIND OF BRINGING
ALL THE DOCTORS FROM
NIH DOWN THERE.

519 01:28:12:05 --:--:--:--
WOW. THERE SHOULD BE 40
PEOPLE WHO WOULD DO THIS.

520 01:28:14:22 01:28:18:06
YEAH. YEAH.

521 01:28:34:10 01:28:36:22
HI, DONNA!

522 01:28:41:28 01:28:43:08
HELLO.

523 01:28:47:20 --:--:--:--
HOLA.

524 01:28:48:21 --:--:--:--
OH!

525 01:28:51:08 --:--:--:--
Donna, voice-over:
THERE'S NOT MUCH MORE
IMPORTANT TO ME THAN FILLING

526 01:28:54:17 --:--:--:--
THIS PROTOCOL.

527 01:28:57:04 --:--:--:--
I VERY MUCH HOPE THAT IT GETS
CLOSER TO BEING FILLED

528 01:29:01:27 01:29:03:16
AT THIS CONFERENCE.

529 01:29:06:11 --:--:--:--
I WANT IT FILLED BECAUSE I
NEED IT FILLED, BUT THE WORTH

530 01:29:10:25 --:--:--:--
OF THIS CONFERENCE IS
MORE THAN JUST FILLING

531 01:29:12:24 --:--:--:--
THAT PROTOCOL.

532 01:29:15:08 --:--:--:--
I'M REALLY HAPPY THAT YOU'RE
HERE, REALLY, BECAUSE THIS IS

533 01:29:17:17 --:--:--:--
THE FIRST TIME THAT YOU COME
TO PUERTO RICO, AND YOU GET TO

534 01:29:19:22 --:--:--:--
EXPERIENCE THE PUERTO
RICAN HOSPITALITY.

535 01:29:24:02 --:--:--:--
Gahl, voice-over:
HERMANSKY-PUDLAK SYNDROME
AFFECTS PATIENTS FROM

536 01:29:26:08 --:--:--:--
PUERTO RICO MORE
THAN FROM ANYWHERE
ELSE IN THE WORLD,

537 01:29:29:24 --:--:--:--
AND THEY'RE DYING.

538 01:29:31:15 --:--:--:--
THIS IS AN INCREDIBLY
MOTIVATING ISSUE FOR US TO TRY

539 01:29:35:13 --:--:--:--
TO DEVELOP A THERAPY.

540 01:29:38:09 --:--:--:--
[WOMAN SPEAKING SPANISH]

541 01:29:39:27 --:--:--:--
Gahl: WE NEED 15
MORE PATIENTS IN ORDER
FOR US TO DO THIS STUDY

542 01:29:43:13 --:--:--:--
AND DETERMINE IF PIRFENIDONE
IS THE TREATMENT THAT SHOULD

543 01:29:46:09 --:--:--:--
BE GIVEN TO ALL HPS PATIENTS
WHO HAVE LUNG DISEASE.

544 01:29:49:24 --:--:--:--
WE'VE DEDICATED ABOUT A DECADE
OF OUR LIVES TOWARDS THIS,

545 01:29:54:15 --:--:--:--
AND WE PLAN TO DEDICATE
MORE TIME AND EFFORT
TOWARDS THIS GOAL.

546 01:30:00:06 --:--:--:--
Donna, voice-over:
DR. GAHL--I REMEMBER HIM
TELLING ME WHEN WE FIRST

547 01:30:02:20 --:--:--:--
STARTED THE PROTOCOL
THAT OFTENTIMES, FOUNDERS

548 01:30:05:23 --:--:--:--
OF DISEASE GROUPS DON'T REAP
THE BENEFITS OF THE FINDINGS.

549 01:30:12:00 --:--:--:--
AND I REMEMBER THINKING,
"WHAT, WAS I, LIKE, SMILING

550 01:30:15:04 --:--:--:--
TOO MUCH TODAY FOR YOU?"

551 01:30:16:19 --:--:--:--
HA HA!

552 01:30:18:16 --:--:--:--
SO HE HAS THIS WONDERFUL SENSE
AND WAY OF BRINGING YOU KIND

553 01:30:21:26 --:--:--:--
OF INTO REALITY THAT,
YOU KNOW, YOU DON'T KNOW

554 01:30:25:27 --:--:--:--
WHETHER WE'LL BE ABLE
TO FIX THIS BY THEN.

555 01:30:29:22 --:--:--:--
AND THAT'S SUCH
A RIGHT THING TO DO.

556 01:30:34:11 --:--:--:--
I THINK OFFERING PEOPLE FALSE
HOPES IS NOT A GOOD THING,

557 01:30:40:21 01:30:47:14
BUT THE HOPE OF THE
SEARCH IS SO NECESSARY.

558 01:30:49:04 01:30:50:21
NICE TO MEET YOU.

559 01:30:55:18 --:--:--:--
Donna, voice-over:
THE THING I CAN'T DO IS TO
GO TO SLEEP AT NIGHT THINKING

560 01:30:59:21 --:--:--:--
THAT I MISSED SOMEBODY
OUT THERE THAT NEEDS US.

561 01:31:04:02 --:--:--:--
I NEED TO FIND THEM.

562 01:31:05:26 --:--:--:--
I NEED TO TRY TO HELP
THEM ALL, AND WE NEED TO

563 01:31:08:25 --:--:--:--
FIND A CURE.

564 01:31:12:05 --:--:--:--
IT'S REALLY QUITE AMAZING AND
REALLY INSPIRATIONAL TO ME TO

565 01:31:15:24 --:--:--:--
SEE THE NUMBER OF PATIENTS,
NOT JUST WITH HPS

566 01:31:18:03 --:--:--:--
BUT WITH OTHER RARE DISORDERS,
WHO COME TO US AND SAY,

567 01:31:22:18 --:--:--:--
"I KNOW THAT NOTHING
CAN BE DONE FOR ME."

568 01:31:25:11 --:--:--:--
THEY LITERALLY SAY THIS,
AND THEY KNOW THEY'RE

569 01:31:27:01 --:--:--:--
GOING TO DIE.

570 01:31:28:16 --:--:--:--
THEY KNOW THEY'RE GOING TO DIE
IN 2 YEARS, OR THEY'RE GOING

571 01:31:30:11 --:--:--:--
TO DIE IN 6 YEARS OR SOME
SUCH, BECAUSE THEY'VE SEEN IT

572 01:31:33:06 --:--:--:--
IN OTHER FAMILY MEMBERS,
AND THEY SAY, "I KNOW THIS IS

573 01:31:35:26 --:--:--:--
"GOING TO HAPPEN, BUT I WANT
TO DO SOMETHING FOR SOMEONE

574 01:31:37:13 --:--:--:--
ELSE," OR, "I WANT TO DO
SOMETHING BECAUSE I KNOW MY

575 01:31:40:20 --:--:--:--
DAUGHTER MIGHT HAVE THIS,
AND SHE'S ONLY 10."

576 01:31:44:03 --:--:--:--
THEY SAY THESE THINGS,
AND IT'S TRUE.

577 01:31:46:21 --:--:--:--
THEY HAVE AN UNDERSTANDING OF
THAT, IN THE SAME SENSE THAT

578 01:31:50:01 --:--:--:--
WE HAVE AN UNDERSTANDING THAT
IF WE HAVE TO SAY GOOD-BYE TO

579 01:31:53:22 --:--:--:--
A PARTICULAR INDIVIDUAL
WITH A DISORDER THAT WE'RE
REALLY HELPING

580 01:32:00:00 --:--:--:--
INDIVIDUALS IN THE
FUTURE OVER THIS.

581 01:32:03:05 --:--:--:--
WE SHOULD START
WITH JUST ASKING HIM
IF HE'S ALREADY

582 01:32:06:16 --:--:--:--
BEEN DIAGNOSED WITH HPS.

583 01:32:08:07 --:--:--:--
[SPEAKING SPANISH]

584 01:32:10:16 --:--:--:--
NO.

585 01:32:11:25 --:--:--:--
WHAT WE WANT TO
TALK ABOUT IS YOUR
PULMONARY FUNCTION

586 01:32:15:21 --:--:--:--
BECAUSE I'M STRONGLY
SUSPICIOUS THAT YOU
DO HAVE HPS.

587 01:32:19:18 01:32:21:12
[SPEAKING SPANISH]

588 01:32:34:01 --:--:--:--
Donna: THIS IS
ABSOLUTELY AMAZING.

589 01:32:35:23 --:--:--:--
Woman:
I KNOW.

590 01:32:37:00 --:--:--:--
I KIND OF FEEL SAD, IN A WAY,
EVEN THOUGH IT'S

591 01:32:39:19 --:--:--:--
SO SUCCESSFUL, BECAUSE THERE'S
SO MUCH I WANT TO GIVE THEM.

592 01:32:42:09 --:--:--:--
SO MANY PEOPLE THAT
NEED HELP.

593 01:32:43:21 --:--:--:--
AND SO MANY PEOPLE
TO TAKE CARE OF.

594 01:32:46:04 --:--:--:--
SO WHILE I'M STANDING HERE
SAYING, "OH, MY GOD, ISN'T

595 01:32:48:07 --:--:--:--
THIS GREAT, WE HAVE SO MANY
PEOPLE," MY HEART IS BREAKING

596 01:32:51:09 01:32:55:13
BECAUSE THERE ARE SO
MANY PEOPLE WITH HPS.

597 01:33:13:08 --:--:--:--
Donna: I'M KIND OF NERVOUS.

598 01:33:14:14 --:--:--:--
WE'RE HERE IN THE BRONX,
TRYING TO HAVE A DAY

599 01:33:18:18 --:--:--:--
OF OUTREACH, AND I
REALLY AM WORRIED.

600 01:33:21:14 --:--:--:--
I HAVE NO IDEA HOW THIS IS
GOING TO WORK, WHETHER IT'S

601 01:33:24:19 --:--:--:--
GOING TO WORK, BUT THIS IS A
NEIGHBORHOOD THAT'S WORTH IT.

602 01:33:27:29 --:--:--:--
IT'S A VERY PUERTO RICAN
NEIGHBORHOOD, AND THIS IS THE

603 01:33:31:18 --:--:--:--
COMMUNITY THAT WE'RE AFTER.

604 01:33:34:23 --:--:--:--
THEN NOBODY EVEN KNOWS
THAT YOU SHORT-SHEETED
YOUR TABLECLOTH.

605 01:33:37:23 --:--:--:--
LOOK AT THAT.

606 01:33:39:06 --:--:--:--
I'M CERTAINLY
PREPARED TO SERVE
BREAKFAST TO ALMOST ALL

607 01:33:42:14 --:--:--:--
OF THE BRONX, AND THERE'S
NOBODY HERE YET, SO I'M KIND

608 01:33:45:29 --:--:--:--
OF WORRIED, AND YOU
JUST HOPE FOR THE BEST.

609 01:33:51:14 --:--:--:--
Donna: OH, HEATHER.

610 01:33:52:29 01:33:54:21
OH, DONNA.

611 01:34:00:20 --:--:--:--
DID YOU GET
A DOUGHNUT?

612 01:34:01:22 --:--:--:--
I WILL NOW.

613 01:34:02:23 --:--:--:--
OK.

614 01:34:03:24 --:--:--:--
HA HA HA!

615 01:34:05:04 --:--:--:--
THERE'S PLENTY.

616 01:34:06:22 --:--:--:--
DO YOU HAVE ANY
HANDOUTS, PAMPHLETS?

617 01:34:08:10 --:--:--:--
SURE.

618 01:34:10:09 --:--:--:--
WE HAVE PAMPHLETS
RIGHT OVER HERE.

619 01:34:12:05 --:--:--:--
HOW ARE YOU?
GOOD MORNING.

620 01:34:13:06 --:--:--:--
GOOD MORNING.

621 01:34:15:05 01:34:17:08
[MANY PEOPLE TALKING AT ONCE]

622 01:34:20:12 --:--:--:--
...BECAUSE HPS IS ONE OF
THE MOST COMMON GENETIC
DISORDERS AMONG PEOPLE

623 01:34:24:13 --:--:--:--
OF PUERTO RICAN
BACKGROUND.

624 01:34:27:19 --:--:--:--
I HAVE A COUSIN.

625 01:34:28:21 --:--:--:--
A COUSIN.

626 01:34:29:21 --:--:--:--
YEAH.
HE'S 24 YEARS OLD.

627 01:34:31:06 --:--:--:--
DOES HE LIVE
IN THE AREA?

628 01:34:33:04 --:--:--:--
HE LIVES IN NEW JERSEY.

629 01:34:35:06 --:--:--:--
AND HE HAS ALBINISM.

630 01:34:36:29 --:--:--:--
YEAH.

631 01:34:38:09 --:--:--:--
DO YOU KNOW ANYTHING
ABOUT HERMANSKY-PUDLAK
SYNDROME?

632 01:34:40:28 --:--:--:--
NO.

633 01:34:42:15 --:--:--:--
IT'S MY FIRST TIME
TO SEE THIS.

634 01:34:44:10 --:--:--:--
OH, FIRST TIME FOR
YOU TO SEE THIS?

635 01:34:47:16 --:--:--:--
ONE OF THE THINGS TO LOOK FOR
IS WHAT'S CALLED NYSTAGMUS,

636 01:34:51:04 --:--:--:--
WHICH IS THIS FUNNY LITTLE
WIGGLING OF THE EYES.

637 01:34:53:04 --:--:--:--
I DON'T KNOW IF YOU CAN SEE,
MY EYES KIND OF HAVE THIS

638 01:34:55:15 --:--:--:--
INVOLUNTARY MOVEMENT.

639 01:34:57:06 --:--:--:--
BECAUSE MY MOM HAS IT.

640 01:34:59:01 --:--:--:--
YOUR MOM HAS HPS?

641 01:35:00:03 --:--:--:--
YES.

642 01:35:01:29 --:--:--:--
[INDISTINCT]

643 01:35:04:11 --:--:--:--
IT'S OK.

644 01:35:05:24 --:--:--:--
ALL MY BROTHER AND
SISTER DON'T HAVE IT.
DON'T HAVE.

645 01:35:10:19 --:--:--:--
DAPPELL@HPSNETWORK.ORG.

646 01:35:13:05 01:35:14:23
LET'S YOU AND ME EMAIL.
OK.

647 01:35:20:05 --:--:--:--
MY HPS HASN'T NECESSARILY
GOTTEN WORSE, BUT I'M HAVING

648 01:35:25:02 --:--:--:--
MORE FREQUENT PROBLEMS,
AND BECAUSE OF THE FREQUENCY

649 01:35:28:15 --:--:--:--
OF THOSE PROBLEMS, I'VE
HAD TO QUIT WORKING.

650 01:35:30:24 --:--:--:--
I QUIT WORKING IN OCTOBER,
AND IT'S NOW MARCH.

651 01:35:35:19 --:--:--:--
IT'S KIND OF FUNNY BECAUSE I
THOUGHT THAT WHEN THE DAY CAME

652 01:35:38:08 --:--:--:--
THAT I'D HAVE TO QUIT WORKING
FOR A WHILE, I THOUGHT I'D BE

653 01:35:41:06 --:--:--:--
REALLY SICK.

654 01:35:42:17 --:--:--:--
I THOUGHT MAYBE I'D BE ON
OXYGEN OR SOMETHING LIKE THAT,

655 01:35:46:05 --:--:--:--
BUT THE SORT OF NAGGING
FREQUENCY OF THE ONGOING

656 01:35:50:26 --:--:--:--
ISSUES EVENTUALLY REALLY
STARTED TO TAKE THEIR TOLL

657 01:35:53:15 --:--:--:--
AT MY JOB.

658 01:35:55:26 --:--:--:--
FOR ONE THING, MISSING SO
MUCH WORK TO GO TO NIH--

659 01:35:59:03 --:--:--:--
EFFECTIVELY, IT WAS COSTING
ME 3 WEEKS OF WORK A YEAR.

660 01:36:03:05 --:--:--:--
PLUS, ON TOP OF THAT, BECAUSE
I'M LEGALLY BLIND, I CAN'T

661 01:36:06:07 --:--:--:--
DRIVE, AND THERE'S NOT
REALLY GREAT PUBLIC TRANSIT

662 01:36:08:26 --:--:--:--
WHERE I LIVE.

663 01:36:10:10 --:--:--:--
SO A 15-MINUTE DOCTOR'S
APPOINTMENT CAN EASILY TAKE ME

664 01:36:13:03 --:--:--:--
AN ENTIRE DAY TO ACCOMPLISH BY
THE TIME I EITHER TAKE A CAB,

665 01:36:16:10 --:--:--:--
TAKE 2 OR 3 BUSES, CHANGE
BUSES TO GET TO AND FROM

666 01:36:19:28 --:--:--:--
A DOCTOR'S APPOINTMENT,
AND I WORKED IN JOURNALISM

667 01:36:22:29 --:--:--:--
AND HAD DEADLINES.

668 01:36:25:12 01:36:26:25
OOPS.

669 01:36:28:27 --:--:--:--
EVENTUALLY, THE WORKLOAD AT
WORK WAS INCREASING, AND I WAS

670 01:36:34:06 --:--:--:--
STRUGGLING TO KEEP UP WITH
WHERE I WAS AND FELT LIKE IT

671 01:36:37:20 --:--:--:--
JUST WASN'T WORKING OUT,
SO I PURSUED GOING

672 01:36:42:02 --:--:--:--
ON DISABILITY.

673 01:36:43:29 --:--:--:--
I'M GOOD.
HOW ARE YOU?

674 01:36:45:04 --:--:--:--
PRETTY GOOD.

675 01:36:46:19 --:--:--:--
Heather, voice-over: I FILED
FOR SOCIAL SECURITY.

676 01:36:48:02 --:--:--:--
I'M STILL WAITING ON THAT.

677 01:36:49:14 --:--:--:--
SO RIGHT NOW, I HAVE NO
INCOME, AND I'M PAYING $450

678 01:36:54:15 --:--:--:--
A MONTH COBRA, PLUS MY
MEDICATIONS RUN BETWEEN $400

679 01:36:59:11 --:--:--:--
AND $600 A MONTH.

680 01:37:01:29 --:--:--:--
SO RIGHT NOW, I'M LIVING ON MY
RETIREMENT SAVINGS, AND I HAVE

681 01:37:06:12 --:--:--:--
ENOUGH MAYBE FOR THE NEXT
MONTH OR TWO, AND THEN...I

682 01:37:11:00 --:--:--:--
DON'T KNOW WHAT WILL HAPPEN.

683 01:37:12:10 01:37:16:19
I'LL HAVE TO MOVE IN WITH
SOME FAMILY OR...SOMETHING.

684 01:37:32:19 --:--:--:--
YOU KNOW, I'M A LITTLE BIT
NERVOUS, JUST BECAUSE I

685 01:37:34:23 --:--:--:--
HAVEN'T SEEN HIM IN A WHILE,
AND IT'S BEEN SINCE

686 01:37:42:10 01:37:44:04
THE SUMMER.

687 01:37:51:22 --:--:--:--
IT'S NOT AS COLD AS I
THOUGHT IT WOULD BE.

688 01:37:53:16 01:37:54:29
NO?

689 01:38:00:25 --:--:--:--
Mervin, voice-over:
WE HAVE A COUPLE
OF THINGS PLANNED.

690 01:38:03:18 --:--:--:--
WE HAVE A MUSICAL PLANNED.

691 01:38:05:10 --:--:--:--
WE HAVE HOPES TO GO TO
A RESTAURANT WHERE

692 01:38:08:08 --:--:--:--
THE WAITERS SING.

693 01:38:10:03 --:--:--:--
Ashley, voice-over: SO
WE'RE GOING TO GO SEE THE
STATUE OF LIBERTY, AND WE'RE

694 01:38:13:04 --:--:--:--
GOING TO DINNER, AND WE'RE
PROBABLY GOING TO GO TO

695 01:38:15:11 --:--:--:--
CENTRAL PARK,
I THINK, TOO.

696 01:38:17:25 --:--:--:--
OH, MY GOSH!
YOU'RE SO COLD!

697 01:38:19:17 --:--:--:--
Mervin, voice-over: I
JUST WANTED TO GIVE THE BOTH
OF US A CHANCE TO GET TO KNOW

698 01:38:22:07 --:--:--:--
EACH OTHER BETTER AND TO
SPEND A LITTLE MORE TIME.

699 01:38:26:18 --:--:--:--
Man: WE WERE CONCERNED ABOUT THE
LOGISTICS OF THE WHOLE THING

700 01:38:28:27 --:--:--:--
AND HOW THEY WERE GOING
TO GET AROUND.

701 01:38:30:29 --:--:--:--
MERVIN THOUGHT HE WAS
JUST GOING TO COME UP HERE

702 01:38:32:18 --:--:--:--
AND WHISK ASHLEY AWAY AND
THE TWO OF THEM DO THE CITY

703 01:38:36:29 --:--:--:--
AND ALL THAT STUFF,
SO WE HAD TO MAKE SOME

704 01:38:38:26 --:--:--:--
ADJUSTMENTS THERE.

705 01:38:40:17 --:--:--:--
WHAT IS THIS?
50th?

706 01:38:42:05 --:--:--:--
YES.

707 01:38:43:25 --:--:--:--
Richard, voice-over:
TWO LEGALLY BLIND PEOPLE
RUNNING AROUND THE CITY COULD

708 01:38:45:11 --:--:--:--
BE A PROBLEM.

709 01:38:47:10 --:--:--:--
[HORN HONKS]

710 01:38:48:14 --:--:--:--
WATCH OUT, WATCH OUT.

711 01:38:50:10 --:--:--:--
Richard, voice-over:
SO WE KIND OF TAILORED THAT
A LITTLE BIT, AND WE'RE KIND

712 01:38:53:03 --:--:--:--
OF AROUND IN THE BACKGROUND.

713 01:38:55:10 --:--:--:--
ARE YOU TAKING
PICTURES OF US?

714 01:38:56:14 --:--:--:--
NO. I'M GOING TO HAVE
A PICTURE OF MYSELF.

715 01:38:59:10 01:39:02:08
WHOO!

716 01:39:07:02 --:--:--:--
Ashley, voice-over:
I'M ABLE TO TALK TO HIM
ABOUT ANYTHING, PRETTY MUCH.

717 01:39:11:07 --:--:--:--
IF WE CONTINUE ON IN OUR
RELATIONSHIP AND EVERYTHING,

718 01:39:14:14 --:--:--:--
YOU KNOW, THERE'S A LOT
OF DECISIONS THAT WE'RE

719 01:39:17:11 --:--:--:--
DEFINITELY GOING TO FACE
IN TERMS OF THINKING

720 01:39:19:20 --:--:--:--
ABOUT WANTING TO
HAVE A CHILD OR NOT.

721 01:39:23:28 --:--:--:--
Mervin, voice-over:
IT'S NOT, WE CAN'T HAVE
CHILDREN, SO WE CAN'T HAVE

722 01:39:27:03 --:--:--:--
A RELATIONSHIP.

723 01:39:28:07 --:--:--:--
IT'S NOT QUITE THAT EXTREME.

724 01:39:30:24 --:--:--:--
IT'S, WE BOTH
HAVE THE SAME HPS.

725 01:39:33:27 --:--:--:--
OK. WHAT ARE WE GOING
TO DO ABOUT IT?

726 01:39:36:00 01:39:37:24
YES.
YES.

727 01:39:41:22 --:--:--:--
Ashley, voice-over:
WE ALWAYS KEEP IT,
LIKE, IN THE BACK OF OUR

728 01:39:44:06 --:--:--:--
MINDS, YOU KNOW?

729 01:39:46:05 --:--:--:--
IT'S JUST GOING THROUGH THE
GOOD AND THE BAD TOGETHER,

730 01:39:49:14 --:--:--:--
AND I THINK IT'S KIND OF
LIKE THE SAME IN ALL OTHER

731 01:39:51:18 --:--:--:--
RELATIONSHIPS, TOO.

732 01:39:54:18 --:--:--:--
HOLD STILL!

733 01:39:55:22 --:--:--:--
I'M TRYING.

734 01:39:56:26 --:--:--:--
OK. THERE. GO.

735 01:39:58:19 01:39:59:27
I GOT IT.

736 01:40:00:27 --:--:--:--
HA HA!

737 01:40:02:16 --:--:--:--
OK.

738 01:40:05:25 --:--:--:--
HOW ARE WE DOING
WITH THE BLOOD?

739 01:40:07:12 --:--:--:--
IT'S GOING VERY WELL.

740 01:40:09:02 --:--:--:--
Gahl, voice-over: WE
NEEDED TO TRY TO GET 40
INDIVIDUALS ENROLLED, AND WE

741 01:40:12:17 --:--:--:--
WANTED TO DO THAT OVER
THE COURSE OF A YEAR.

742 01:40:14:16 --:--:--:--
IT TURNS OUT IT TOOK US
WELL OVER 3 YEARS TO GET 35

743 01:40:18:17 01:40:20:29
INDIVIDUALS INVOLVED.

744 01:40:23:25 --:--:--:--
BUT WE WERE ALLOWED TO DO AN
INTERIM ANALYSIS OF THE DATA,

745 01:40:27:10 --:--:--:--
HOW WELL WERE THE PATIENTS
DOING, APPROXIMATELY ONE YEAR

746 01:40:31:08 --:--:--:--
AFTER 30 INDIVIDUALS HAD BEEN
ENROLLED, AND THAT INTERIM

747 01:40:34:28 --:--:--:--
ANALYSIS INDICATED THERE WOULD
NEVER BE A DIFFERENCE BETWEEN

748 01:40:41:01 --:--:--:--
THE PIRFENIDONE-TREATED AND
THE NON-TREATED INDIVIDUALS,

749 01:40:45:03 --:--:--:--
AND FUTILITY IS A CAUSE
TO STOP THE TRIAL.

750 01:40:50:14 --:--:--:--
WHEN WE SAW THOSE
NUMBERS, IT WAS,

751 01:40:52:20 --:--:--:--
IN MANY RESPECTS,
HEARTBREAKING--NOT JUST

752 01:40:55:02 01:40:58:10
HEARTBREAKING FOR ME,
BUT FOR OUR ENTIRE TEAM.

753 01:41:00:21 --:--:--:--
I HAD TO MAKE A CALL TO
DONNA TO TELL HER THAT.

754 01:41:05:03 --:--:--:--
I DIDN'T KNOW EXACTLY HOW
I WAS GOING TO DO THIS.

755 01:41:09:03 --:--:--:--
WHEN I GOT THE CALL AND
LISTENED TO WHAT DR. GAHL SAID,

756 01:41:12:08 01:41:16:19
I WAS KIND OF, LIKE, JUST
KIND OF NOT PREPARED.

757 01:41:19:16 --:--:--:--
I GUESS THE THING THAT HIT
ME THE MOST WAS, WHAT

758 01:41:22:13 --:--:--:--
ABOUT THE PEOPLE WHO ARE BEING
TREATED WITH PIRFENIDONE,

759 01:41:25:13 --:--:--:--
AND THEN THINKING ABOUT
THE PEOPLE WHO ARE JUST

760 01:41:27:22 --:--:--:--
IN THE TRIAL, AND EVEN IF
THEY'RE ON PLACEBO, I THINK

761 01:41:31:14 --:--:--:--
THAT, YOU KNOW, THAT HOPE
IS MEDICATION IN ITSELF.

762 01:41:36:03 --:--:--:--
JUST BEING PART OF
THE TRIAL IS HELPFUL.

763 01:41:38:25 --:--:--:--
SO, YOU KNOW, JUST THE THOUGHT
OF HAVING TO TELL EVERYBODY

764 01:41:44:08 --:--:--:--
AND HAVE THE DRUG TAKEN AWAY
WAS JUST HEART-WRENCHING.

765 01:41:49:25 --:--:--:--
WE HAD TO GO THROUGH THIS
BECAUSE PIRFENIDONE HAD TO BE

766 01:41:52:15 --:--:--:--
TESTED IN THIS GROUP
OF INDIVIDUALS.

767 01:41:55:23 --:--:--:--
SO ONE HAS TO TAKE THE
PHILOSOPHICAL VIEW, OR ELSE

768 01:42:00:10 --:--:--:--
ONE BECOMES REASONABLY
DEPRESSED ABOUT CONTINUING.

769 01:42:04:21 --:--:--:--
AND I HAD TO ACTUALLY ASK HIM--

770 01:42:07:14 --:--:--:--
AND I WAS SO SCARED TO--

771 01:42:09:05 --:--:--:--
I'M LIKE, WHAT DOES THIS
MEAN FOR OUR FUTURE

772 01:42:10:29 --:--:--:--
AND DRUG THERAPY?

773 01:42:13:13 --:--:--:--
AND I GOT PRETTY MUCH THE
ANSWER I WAS EXPECTING,

774 01:42:17:23 --:--:--:--
BUT NOT EXACTLY THE
ANSWER I WANTED.

775 01:42:21:14 --:--:--:--
WE'RE NOT REALLY AT A STAGE
WHERE WE CAN PROPOSE ANOTHER

776 01:42:25:23 --:--:--:--
CLINICAL TRIAL--AT LEAST
I DON'T FEEL THAT WAY.

777 01:42:28:20 --:--:--:--
WE DON'T HAVE ENOUGH
PROSPECTS IN THAT REGARD.

778 01:42:33:00 --:--:--:--
AND THAT WAS A KILLER.

779 01:42:36:07 --:--:--:--
BUT HE TRULY COMFORTED ME
BY SAYING, WE'RE NOT

780 01:42:40:04 --:--:--:--
TURNING OUR BACK.

781 01:42:41:12 --:--:--:--
THIS KIND OF GIVES US MORE
TIME TO REFOCUS OUR ENERGY

782 01:42:46:08 --:--:--:--
IN THE LABORATORY AND LOOK
FOR A MORE FOCUSED THERAPY.

783 01:42:50:02 --:--:--:--
I KNOW YOUR LEGS MUST BE
KILLING YOU RIGHT NOW.

784 01:42:51:25 --:--:--:--
YOU'RE DOING FINE,
THOUGH.

785 01:42:53:02 --:--:--:--
YOU'RE DOING GREAT.

786 01:42:54:04 01:42:56:00
TRY TO KEEP IT UP
IF YOU CAN.

787 01:42:58:16 --:--:--:--
Heather, voice-over:
IT DID CATCH US COMPLETELY
BY SURPRISE, AND I

788 01:43:01:02 --:--:--:--
WAS DEVASTATED.

789 01:43:02:11 --:--:--:--
FOR LIKE 3 DAYS, I DIDN'T
WANT TO GET OUT OF BED.

790 01:43:04:04 --:--:--:--
IT WAS JUST...IT WAS
TOTALLY DEVASTATING.

791 01:43:08:04 --:--:--:--
[TELEPHONE RINGS]

792 01:43:09:06 --:--:--:--
HELLO.

793 01:43:10:07 --:--:--:--
HI, HEATHER.

794 01:43:11:07 --:--:--:--
IT'S KEVIN O'BRIEN FROM NIH.

795 01:43:12:07 --:--:--:--
HEY. HOW ARE
YOU DOING?

796 01:43:13:29 --:--:--:--
OK. GOOD. YOU?

797 01:43:15:02 --:--:--:--
I'M GREAT.

798 01:43:16:16 --:--:--:--
HEATHER, I'M CALLING YOU TO
UPDATE YOU ON THE PIRFENIDONE

799 01:43:19:04 --:--:--:--
TRIAL AND ALSO TO REVEAL YOUR
TREATMENT GROUP ASSIGNMENT.

800 01:43:23:02 --:--:--:--
YOU HAD A TWO-THIRD CHANCE
OF BEING ON PIRFENIDONE

801 01:43:26:02 --:--:--:--
AND A ONE-THIRD CHANCE
OF BEING ON PLACEBO.

802 01:43:30:00 --:--:--:--
HEATHER, YOU WERE
RANDOMIZED TO RECEIVE PLACEBO.

803 01:43:33:26 --:--:--:--
OH, WOW.

804 01:43:35:12 --:--:--:--
AND THAT IS WHAT YOU'VE BEEN
TAKING SINCE THE BEGINNING

805 01:43:37:29 --:--:--:--
OF THE TRIAL.

806 01:43:40:20 --:--:--:--
OK.

807 01:43:41:29 --:--:--:--
SO KEVIN CALLED ME
THIS AFTERNOON FROM NIH,
AND I WAS

808 01:43:45:02 --:--:--:--
INDEED ON PLACEBO
THE WHOLE TIME.

809 01:43:47:01 --:--:--:--
REALLY?

810 01:43:48:11 --:--:--:--
OH, INTERESTING.

811 01:43:49:28 --:--:--:--
SO WHAT DOES THAT MEAN?

812 01:43:51:14 --:--:--:--
THAT MEANS I WAS TAKING A
SUGAR PILL THE WHOLE TIME.

813 01:43:53:15 --:--:--:--
HA HA HA!

814 01:43:54:15 --:--:--:--
I KNOW THAT, BUT
DOES THAT MEAN--

815 01:43:55:15 --:--:--:--
I THOUGHT I WAS ON IT.

816 01:43:57:23 --:--:--:--
I REALLY DID.

817 01:43:59:13 --:--:--:--
YEAH, YEAH.

818 01:44:02:04 --:--:--:--
DOES THAT MAKE YOU FEEL
KIND OF PISSED, THOUGH?

819 01:44:04:13 --:--:--:--
NOT REALLY BECAUSE THE WAY
I KIND OF FIGURE IT IS,

820 01:44:09:22 --:--:--:--
I'VE BEEN PRETTY STABLE.

821 01:44:11:03 --:--:--:--
MY FUNCTIONS WENT UP
WHEN I WAS THINKING
I WAS GETTING THE

822 01:44:14:17 --:--:--:--
DRUG, SO THAT MAKES
ME WONDER WHAT THAT
WAS ALL ABOUT,

823 01:44:17:03 --:--:--:--
BUT THEY'VE FALLEN AGAIN,
BUT THEY'VE FALLEN TO WHERE

824 01:44:20:19 --:--:--:--
THEY WERE WHEN I ENTERED THE
TRIAL, SO TECHNICALLY, I'M

825 01:44:22:19 --:--:--:--
IN THE SAME SPOT I WAS IN 3
YEARS AGO, WHICH WE DIDN'T

826 01:44:26:10 --:--:--:--
THINK WAS GOING TO HAPPEN.

827 01:44:27:18 --:--:--:--
WE THOUGHT IT WOULD PROGRESS,
SO THAT'S GOOD.

828 01:44:31:00 --:--:--:--
SHALL WE POP
ONE OPEN?

829 01:44:32:11 --:--:--:--
YEAH. LET'S POP ONE OPEN.

830 01:44:33:18 --:--:--:--
HA HA!

831 01:44:34:25 --:--:--:--
NO. I DON'T
KNOW WHAT
IT IS. IT'S SILLY.

832 01:44:36:13 --:--:--:--
THAT'S WHAT WAS IN IT.

833 01:44:37:14 --:--:--:--
TASTE IT.

834 01:44:40:16 --:--:--:--
UH-OH.

835 01:44:42:28 --:--:--:--
TASTES LIKE FLOUR TO ME.

836 01:44:43:28 --:--:--:--
I DON'T TASTE ANYTHING.

837 01:44:45:05 --:--:--:--
MAYBE THERE'S BAKING SODA
IN THERE OR SOMETHING.

838 01:44:47:21 --:--:--:--
I GUESS IT REALLY WAS
ALL IN MY HEAD.

839 01:44:49:03 --:--:--:--
HA HA HA!

840 01:45:01:13 --:--:--:--
Donna: SO, HERE WE GO.

841 01:45:02:27 --:--:--:--
THIS IS THE 17th
ANNUAL CONFERENCE.

842 01:45:05:07 --:--:--:--
CAN YOU BELIEVE IT?

843 01:45:06:11 --:--:--:--
17 YEARS WE'VE BEEN
DOING THIS SHINDIG.

844 01:45:08:29 01:45:11:03
[APPLAUSE]

845 01:45:16:08 --:--:--:--
SO WE HAVE A THEME
EVERY YEAR, RIGHT?

846 01:45:20:10 --:--:--:--
AND THIS YEAR OUR THEME IS
TO BELIEVE IN YOUR DREAMS--

847 01:45:23:29 --:--:--:--
OUR DREAM OF NIH AND THE CURE,
AND IT'S ALL OF THE DREAMS

848 01:45:28:04 --:--:--:--
THAT WE THINK ABOUT IN OUR
COMMUNITY, SO IT'S IMPORTANT

849 01:45:32:16 --:--:--:--
TO BELIEVE IN YOUR DREAMS,
AND THAT'S WHAT WE'RE GOING TO

850 01:45:35:26 --:--:--:--
CELEBRATE THIS NEXT
COUPLE OF DAYS.

851 01:45:39:10 --:--:--:--
THE HPS RESEARCH
PIRFENIDONE TRIAL--

852 01:45:42:28 --:--:--:--
I'D HOPED TO KIND OF BE SAD
FOR A MINUTE AND FEEL BAD

853 01:45:47:02 --:--:--:--
ABOUT IT A LITTLE
BIT AND MOVE ON.

854 01:45:52:20 --:--:--:--
AND QUITE FRANKLY, IF I
WAS ONE OF THE PERSONS

855 01:45:56:25 --:--:--:--
ON THE TRIAL TAKING THE DRUG,
I'M NOT EVEN EXACTLY SURE

856 01:46:01:06 --:--:--:--
HOW I'D FEEL.

857 01:46:04:11 --:--:--:--
AND I CONGRATULATE, COMMEND,
AND I'M SO IN AWE

858 01:46:11:04 --:--:--:--
OF THE PEOPLE WHO WERE IN THAT
TRIAL, WHO WORKED REALLY HARD,

859 01:46:15:07 --:--:--:--
AND THANK YOU ON BEHALF OF MY
FAMILY AND THE HPS NETWORK.

860 01:46:19:27 --:--:--:--
GOD BLESS YOU.

861 01:46:20:29 01:46:23:02
[APPLAUSE]

862 01:46:28:09 01:46:31:05
[SPEAKING SPANISH]

863 01:46:45:19 --:--:--:--
I MET MY HUSBAND HERE AT
THE CONFERENCE, AND WE

864 01:46:47:16 --:--:--:--
GOT MARRIED.

865 01:46:48:26 01:46:50:20
[SPEAKING SPANISH]

866 01:46:53:00 --:--:--:--
SO THOSE OF YOU WHO ARE
SINGLE, FEMALES AND MALES,

867 01:46:57:06 --:--:--:--
YOU KNOW, YOU CAN
MEET SOMEBODY HERE.

868 01:46:59:01 --:--:--:--
HA HA!

869 01:47:00:09 --:--:--:--
[APPLAUSE]

870 01:47:02:02 --:--:--:--
MY MAJOR PORTION LAST YEAR WAS
A LUNG TRANSPLANT.

871 01:47:05:22 --:--:--:--
UP TO THAT POINT, I'D BEEN ON
OXYGEN 24/7, AND IT TOOK ME,

872 01:47:12:15 --:--:--:--
LIKE, 2 MINUTES TO CLIMB
THE STEPS UP TO MY BEDROOM

873 01:47:14:26 --:--:--:--
AT NIGHT, SO...YOU KNOW,
FOLLOWING AFTER 14

874 01:47:18:25 --:--:--:--
GRANDCHILDREN, I THINK I
SHOULD GET INTO SHAPE.

875 01:47:21:03 --:--:--:--
HA HA!

876 01:47:22:08 --:--:--:--
[APPLAUSE]

877 01:47:25:07 --:--:--:--
I'M VERY HAPPY TO SAY
THAT ONE OF THE MOST
SIGNIFICANT THINGS

878 01:47:28:22 --:--:--:--
THAT HAPPENED IN
MY LIFE LAST YEAR WAS
THAT I GOT TO KNOW

879 01:47:31:07 --:--:--:--
THIS YOUNG LADY
A LOT BETTER.

880 01:47:32:24 --:--:--:--
[APPLAUSE]

881 01:47:36:07 --:--:--:--
ù TILL NOW I ALWAYS GOT
BY ON MY OWN ù

882 01:47:41:29 --:--:--:--
ù I NEVER REALLY CARED ù

883 01:47:44:05 --:--:--:--
ù UNTIL I MET YOU ù

884 01:47:48:01 --:--:--:--
ù AND NOW IT CHILLS ME
TO THE BONE ù

885 01:47:51:21 --:--:--:--
ù HOW DO I
GET YOU ALONE? ù

886 01:47:57:12 --:--:--:--
ù HOW DO I GET
YOU ALONE? ù

887 01:48:00:24 --:--:--:--
[APPLAUSE AND CHEERING]

888 01:48:02:24 01:48:05:09
[MUSIC PLAYING]

889 01:48:29:07 --:--:--:--
ù I'M TOO SEXY FOR MY LUNGS,
TOO SEXY FOR MY LUNGS LUNGS ù

890 01:48:33:20 01:48:35:29
ù GOING TO LEAVE ME ù

891 01:48:37:04 --:--:--:--
WHOO HOO!

892 01:48:38:10 --:--:--:--
WILSON STARTED TEASING ME
ABOUT DOING THIS DANCE,

893 01:48:41:06 --:--:--:--
AND SO FINALLY I SAID,
ALL RIGHT, BUDDY, IF YOU GET

894 01:48:43:09 --:--:--:--
YOUR LUNG TRANSPLANT AND MAKE
IT TO THE CONFERENCE NEXT

895 01:48:45:09 --:--:--:--
YEAR, I'LL DO THE DANCE.

896 01:48:47:00 --:--:--:--
WHOO!

897 01:48:48:06 01:48:49:17
[MUSIC PLAYING]

898 01:49:00:29 --:--:--:--
Woman: ONE OF
THE OTHER HPSers SAID,
IF YOU DO IT, I'LL GIVE $100

899 01:49:05:24 --:--:--:--
TO THE NETWORK, SO, KNOWING
THE FUNDRAISER SHE IS--

900 01:49:10:13 --:--:--:--
YEAH. I'LL DO
ANYTHING FOR MONEY.

901 01:49:12:11 --:--:--:--
HA HA HA!

902 01:49:13:27 --:--:--:--
NOT REALLY.

903 01:49:15:27 01:49:17:23
WHOO!

904 01:49:20:23 --:--:--:--
ù I'M TOO SEXY FOR MY LUNGS,
TOO SEXY FOR MY LUNGS LUNGS ù

905 01:49:25:06 --:--:--:--
ù GOING TO LEAVE ME ù

906 01:49:27:07 --:--:--:--
[MUSIC ENDS]

907 01:49:28:19 01:49:30:21
[APPLAUSE AND CHEERING]

908 01:49:39:27 --:--:--:--
LOOK AT THAT, HEATHER.

909 01:49:40:28 --:--:--:--
YOU'RE GETTING A
STANDING OVATION!

910 01:49:44:26 --:--:--:--
Heather, voice-over:
WHEN YOU FIRST GET INVOLVED,
YOU'RE TRYING TO CURE

911 01:49:47:06 --:--:--:--
YOURSELF, AND YOU'RE LOOKING
OUT--NOT THAT YOU DON'T WANT

912 01:49:50:20 --:--:--:--
TO HELP ANYBODY ELSE,
BUT YOU'RE VERY CONCERNED

913 01:49:52:21 --:--:--:--
ABOUT YOUR OWN HEALTH, AND
AS YOU GET MORE INVOLVED

914 01:49:54:17 --:--:--:--
IN THE RESEARCH AND MORE
CONNECTED TO THE COMMUNITY,

915 01:49:58:12 --:--:--:--
IT BECOMES NOT JUST ABOUT YOU,
BUT ABOUT EVERYBODY ELSE,

916 01:50:01:13 --:--:--:--
AND I THINK EVERYBODY
FEELS THAT WAY.

917 01:50:04:06 01:50:05:23
THANK YOU.

918 01:50:07:23 --:--:--:--
WHAT WE WON'T
DO...

919 01:50:09:00 --:--:--:--
FOR THE CURE.

920 01:50:10:10 --:--:--:--
THAT'S RIGHT.

921 01:50:11:10 01:50:13:15
HA HA HA!

922 01:50:14:21 --:--:--:--
HELLO, EVERYBODY.
GOOD EVENING.

923 01:50:16:09 --:--:--:--
I DON'T LIKE SPEAKING IN
PUBLIC, BUT I ACTUALLY ASKED

924 01:50:19:16 --:--:--:--
TO DO THIS TONIGHT BECAUSE
TO ME, IT'S VERY IMPORTANT

925 01:50:22:17 --:--:--:--
FOR DR. GAHL.

926 01:50:25:25 --:--:--:--
I WANTED TO SPEAK TO HIM.

927 01:50:27:19 --:--:--:--
I AM THE FIRST PERSON TO
OPEN THE PROTOCOL AT THE NIH.

928 01:50:32:25 --:--:--:--
HE'S RIGHT IN FRONT
OF YOU.

929 01:50:35:11 --:--:--:--
AND I ACTUALLY--THE DRUG TRIAL
ENDED, AND THEY CALLED ME,

930 01:50:42:12 --:--:--:--
AND I WAS VERY SURPRISED
ON THE PHONE.

931 01:50:44:22 --:--:--:--
AND I WANTED YOU TO KNOW HOW
GRATEFUL I AM THAT YOU DID THE

932 01:50:50:09 --:--:--:--
PROTOCOL FOR ME, AND I WANTED
YOU AND THE NIH TO KNOW HOW

933 01:50:54:10 --:--:--:--
MUCH I APPRECIATED IT.

934 01:50:56:02 --:--:--:--
AND WHEN I HEARD THAT
THE PROTOCOL WAS CLOSED,

935 01:50:58:21 --:--:--:--
THAT NIGHT, INSTEAD
OF THINKING OF ME, I

936 01:51:01:17 --:--:--:--
THOUGHT OF YOU.

937 01:51:02:27 --:--:--:--
I SAID I WANTED DR. GAHL TO
KNOW THAT HE DID NOT LET ME

938 01:51:05:22 --:--:--:--
DOWN, AND THAT I KNOW THAT
EVEN THOUGH THE PROTOCOL HAS

939 01:51:09:29 --:--:--:--
ENDED, STILL HE'S
MY DREAM-MAKER.

940 01:51:13:14 --:--:--:--
HE'S THE PERSON...

941 01:51:14:15 --:--:--:--
[APPLAUSE]

942 01:51:15:29 --:--:--:--
THAT IS MY DREAM-MAKER,
AND YOU ARE MY HOPE.

943 01:51:21:06 --:--:--:--
THE PROTOCOL WASN'T MY HOPE.

944 01:51:23:28 --:--:--:--
YOU ARE MY HOPE, AND THE
NIH IS MY HOPE, AND I AM

945 01:51:28:10 --:--:--:--
SO BLESSED TO HAVE
YOU IN MY LIFE.

946 01:51:33:13 --:--:--:--
THIS IS PRESENTED TO YOU.

947 01:51:34:26 01:51:37:13
[APPLAUSE]

948 01:51:43:24 --:--:--:--
Gahl, voice-over: PATIENTS
AND GROUPS--IN A WAY,
THEY REALLY DON'T WANT YOU TO

949 01:51:47:18 --:--:--:--
CRY WITH THEM.

950 01:51:48:29 --:--:--:--
THEY WANT YOU TO BE THE STRONG
PERSON BECAUSE THEY KNOW THAT

951 01:51:53:16 --:--:--:--
YOU HAVE TO GO FORWARD.

952 01:51:54:19 01:51:56:24
SOMEBODY HAS TO GO FORWARD.

953 01:52:05:18 --:--:--:--
I TOOK HER TO THE
PEDIATRICIAN, AND I SAID,

954 01:52:07:17 --:--:--:--
"SHE'S GOT THESE BRUISES,"
AND HE SAID, "SHE'S ALBINO.

955 01:52:10:02 --:--:--:--
IT'S VISION.
IT'S SKIN.

956 01:52:11:14 --:--:--:--
GO AND BE WELL."

957 01:52:13:06 --:--:--:--
I BROUGHT HER BACK AGAIN.

958 01:52:14:14 --:--:--:--
"THESE BRUISES ARE--SOMETHING
IS WRONG WITH THIS."

959 01:52:16:29 --:--:--:--
AND HE SAID, "YOU'RE
A NURSE MOTHER.

960 01:52:19:14 --:--:--:--
"STOP WORRYING.
STOP READING. JUST GO."

961 01:52:22:26 --:--:--:--
Donna, voice-over:
I'VE BEEN DOING THIS FOR AN
AWFULLY LONG TIME, AND I'M

962 01:52:26:00 --:--:--:--
STILL GOING TO A CLINIC IN THE
BRONX, WHERE THERE ARE SO MANY

963 01:52:30:12 --:--:--:--
PUERTO RICAN PEOPLE,
AND PHYSICIANS HAVE NEVER

964 01:52:33:08 --:--:--:--
HEARD OF THIS.

965 01:52:34:20 --:--:--:--
I MEAN,
IT'S ALMOST SAD, IN A WAY,

966 01:52:37:03 --:--:--:--
BECAUSE YET AGAIN, IT PROVES
TO ME THAT WE HAVE SO MANY

967 01:52:40:07 01:52:42:01
MOUNTAINS TO CLIMB.

968 01:52:48:04 --:--:--:--
ALBINISM.

969 01:52:49:23 --:--:--:--
PEOPLE WITH ALBINISM HAVE
VERY LIGHT SKIN, LIKE ME.

970 01:52:53:06 --:--:--:--
Donna, voice-over:
MY FEELING IS THAT THE
PROTOCOL WAS PRETTY HARD TO

971 01:52:56:07 --:--:--:--
FILL, AND THE NEXT TIME WE
EVER HAVE THAT OPPORTUNITY,

972 01:52:59:11 --:--:--:--
I WANT TO BE DOUBLE THE SIZE.

973 01:53:01:10 --:--:--:--
I NEED TO DOUBLE THE SIZE OF
THIS ORGANIZATION, AND I WANT

974 01:53:03:24 --:--:--:--
TO DO IT NOW.

975 01:53:05:13 --:--:--:--
SO IT'S KIND OF, LIKE, WILD TO
THINK THAT ANYTHING IS GOING

976 01:53:10:05 --:--:--:--
TO COME OUT OF THIS OR THAT
IT'S PRACTICAL IN ANY WAY,

977 01:53:13:09 --:--:--:--
SHAPE, OR FORM, BUT WHO SAYS
TRYING TO CURE A RARE DISEASE

978 01:53:16:29 --:--:--:--
IS ANYTHING--IS PRACTICAL?

979 01:53:19:14 01:53:23:04
I MEAN, IT'S AN
IMPOSSIBLE DREAM AT BEST.

980 01:53:24:04 --:--:--:--
SO WHY NOT?

981 01:53:25:24 01:53:29:01
LET'S JUST TRY, AND
HOPE THAT IT WORKS.

982 01:54:58:13 --:--:--:--
CAPTIONING MADE POSSIBLE BY
FRIENDS OF NCI

983 01:55:01:15 --:--:--:--
CAPTIONED BY THE NATIONAL
CAPTIONING INSTITUTE
--www.ncicap.org--

984 01:55:04:15 01:55:05:15