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Any news regarding PTC124?

M

miesl

New member
I haven't seen anything recently - but have you joined the mailing list on the PTC website? It looks like they mail out updates when they have them.

I don't know much about clinical studies with infants since they are limited on quality of life feedback and spirometry - but I would mention to your CF doc that you're interested in participating in studies if your daughter qualifies. I would assume that based on the favorable Phase 2 studies, PTC will be moving to Phase 3 soon and need more people for testing.

Studies are wonderful to participate in! Not only can you get experimental meds and treatment options - some of them pay quite nicely.

-Michelle
 
M

miesl

New member
I haven't seen anything recently - but have you joined the mailing list on the PTC website? It looks like they mail out updates when they have them.

I don't know much about clinical studies with infants since they are limited on quality of life feedback and spirometry - but I would mention to your CF doc that you're interested in participating in studies if your daughter qualifies. I would assume that based on the favorable Phase 2 studies, PTC will be moving to Phase 3 soon and need more people for testing.

Studies are wonderful to participate in! Not only can you get experimental meds and treatment options - some of them pay quite nicely.

-Michelle
 
M

miesl

New member
I haven't seen anything recently - but have you joined the mailing list on the PTC website? It looks like they mail out updates when they have them.

I don't know much about clinical studies with infants since they are limited on quality of life feedback and spirometry - but I would mention to your CF doc that you're interested in participating in studies if your daughter qualifies. I would assume that based on the favorable Phase 2 studies, PTC will be moving to Phase 3 soon and need more people for testing.

Studies are wonderful to participate in! Not only can you get experimental meds and treatment options - some of them pay quite nicely.

-Michelle
 
M

miesl

New member
I haven't seen anything recently - but have you joined the mailing list on the PTC website? It looks like they mail out updates when they have them.

I don't know much about clinical studies with infants since they are limited on quality of life feedback and spirometry - but I would mention to your CF doc that you're interested in participating in studies if your daughter qualifies. I would assume that based on the favorable Phase 2 studies, PTC will be moving to Phase 3 soon and need more people for testing.

Studies are wonderful to participate in! Not only can you get experimental meds and treatment options - some of them pay quite nicely.

-Michelle
 
M

miesl

New member
I haven't seen anything recently - but have you joined the mailing list on the PTC website? It looks like they mail out updates when they have them.
<br />
<br />I don't know much about clinical studies with infants since they are limited on quality of life feedback and spirometry - but I would mention to your CF doc that you're interested in participating in studies if your daughter qualifies. I would assume that based on the favorable Phase 2 studies, PTC will be moving to Phase 3 soon and need more people for testing.
<br />
<br />Studies are wonderful to participate in! Not only can you get experimental meds and treatment options - some of them pay quite nicely.
<br />
<br />-Michelle
 
N

NoExcuses

New member
Marla you have probably seen this before but the CFF updates this pipeline regularly <img src="i/expressions/face-icon-small-smile.gif" border="0">


<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/DrugDevelopmentPipeline/">http://www.cff.org/research/DrugDevelopmentPipeline/</a>
 
N

NoExcuses

New member
Marla you have probably seen this before but the CFF updates this pipeline regularly <img src="i/expressions/face-icon-small-smile.gif" border="0">


<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/DrugDevelopmentPipeline/">http://www.cff.org/research/DrugDevelopmentPipeline/</a>
 
N

NoExcuses

New member
Marla you have probably seen this before but the CFF updates this pipeline regularly <img src="i/expressions/face-icon-small-smile.gif" border="0">


<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/DrugDevelopmentPipeline/">http://www.cff.org/research/DrugDevelopmentPipeline/</a>
 
N

NoExcuses

New member
Marla you have probably seen this before but the CFF updates this pipeline regularly <img src="i/expressions/face-icon-small-smile.gif" border="0">


<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/DrugDevelopmentPipeline/">http://www.cff.org/research/DrugDevelopmentPipeline/</a>
 
N

NoExcuses

New member
Marla you have probably seen this before but the CFF updates this pipeline regularly <img src="i/expressions/face-icon-small-smile.gif" border="0">
<br />
<br />
<br /><a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/DrugDevelopmentPipeline/">http://www.cff.org/research/DrugDevelopmentPipeline/</a>
 
F

Faust

New member
My thoughts regarding PTC124: This will be wonderful news for the smaller percentage of CF's with mutations ending in X (from what I have been told, those are the only nonsense mutations). For the others without these mutations, it will mean nothing besides knowing others are technically being cured.
 
F

Faust

New member
My thoughts regarding PTC124: This will be wonderful news for the smaller percentage of CF's with mutations ending in X (from what I have been told, those are the only nonsense mutations). For the others without these mutations, it will mean nothing besides knowing others are technically being cured.
 
F

Faust

New member
My thoughts regarding PTC124: This will be wonderful news for the smaller percentage of CF's with mutations ending in X (from what I have been told, those are the only nonsense mutations). For the others without these mutations, it will mean nothing besides knowing others are technically being cured.
 
F

Faust

New member
My thoughts regarding PTC124: This will be wonderful news for the smaller percentage of CF's with mutations ending in X (from what I have been told, those are the only nonsense mutations). For the others without these mutations, it will mean nothing besides knowing others are technically being cured.
 
F

Faust

New member
My thoughts regarding PTC124: This will be wonderful news for the smaller percentage of CF's with mutations ending in X (from what I have been told, those are the only nonsense mutations). For the others without these mutations, it will mean nothing besides knowing others are technically being cured.
<br />
<br />
<br />
 
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