Premature Stop Codon Discussions (12)

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Can someone who's boy is getting Ataluren / PTC124 speak to the benefits?

Hi,   I am eager for information from any / all parents who's boys have been part of the Ataluren trials.  Can you speak to your perception…

Started by John Smith

3 Jan 26, 2012
Reply by John Smith

Any word on potential FDA approval date for Ataluren / PTC 124 ?

Hello,   Anybody out there?   Was wondering if anyone had any word on a possible Ataluren / PTC 124 approval time frame.  Or, if anyone has…

Started by John Smith

1 Sep 2, 2011
Reply by Chad Studebaker

Ataluren - PTC124

Bob,   Jacob was on the drug high dose for over a year.  We felt like he had more mobility and energy when taking the drug. Not sure if thi…

Started by Chad Studebaker

3 Dec 13, 2010
Reply by John Smith

Vision problems?

Has anyone's son had vision problems that came to light from a yearly school check?  Depth, ocular motor, etc.?We've noticed he has a diffi…

Started by Michelle

0 Feb 8, 2010

PTC124 and steroids

Hi there, I was just wondering if any of the boys enrolled in the current Phase 2b trial are not taking steroids - and whether any of the…

Started by Marks33

1 Jul 1, 2009
Reply by Perlita & Gordy Hains

Tried to enroll our son into the PTC124/2b trial - no luck so far - so frustrated

Hi all My name is Berit from Norway, and we have a 9 year old boy with a "nonsense" mutation. He fits ALL the inclusion criteria to partic…

Started by BeritSofie

2 Nov 29, 2008
Reply by BeritSofie

124b extension details?

We are currently considering whether to enrol our son in the PTC124b clinical trial. He was not in the phase 2a. Does anyone know (based o…

Started by brad stringer

3 Oct 17, 2008
Reply by brad stringer


Hi there, I note from the Clinical Trials website there is a 6 month phase 1 trial for DMD with stop codons, currently recruiting, for Gen…

Started by Marks33

1 Sep 7, 2008
Reply by Tammy Gregory

Recent PTC Litigation

In case you have not seen the article, be aware that a parent of a stop-codon child has filed suit against PTC Theraputics. Obviously we ar…

Started by Michelle

1 Aug 21, 2008
Reply by Tamara Walters

PTC124 upcoming Trial and its requirements...

My son turns 5 in October and we hope to have him screened as a possible participant for the PTC124 study here at CHoP at that time. My wif…

Started by Paul Johnson

7 Aug 21, 2008
Reply by Tamara Walters


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