ATALUREN - confused about the information given from PTC Therapeutics and Genzyme


Hi Sharon and Pat

or anyone else who can help


Can someone PLEASE help me understand the information given from PTC Therapeutics and Genzyme, regards to Ataluren. Our son can use Ataluren, and he is about to stop walking. He didn’t participate in the
previous clinical trials, because the enrollment was closed before our son
could enter the trial. I know there are many other boys ”out there” in the same

I am asking for several other parents coming from Norway,but also parents coming from other contries.


First we had this article from the Annual Meeting of the American Academy of Neurology, published

April 13th. This article didn’t say anything that this is going to be a follow-on clinical study of Ataluren, so I thought ohhhhh finally Ataluren will be on the market in the USA (if approved by the FDA):



AAN 2011: Ataluren Reduces Decline in Ambulation in Duchenne Muscular Dystrophy

Based on these results, PTC Therapeutics, the company that developed ataluren, is planning to seek approval for its use in DMD from the United States Food and Drug Administration. Because of
the mechanism by which the drug overcomes the effects of the mutation, it would
be appropriate for only about 13% of DMD patients, according to study presenter
Ted Abresch, Research Associate in Neuromuscular Disease at the University of
California at Davis.


The whole article published April 13th 2011


Then today, this article was publised by Genzyme. They are talking about a follow-on open label clinical study for previous trial participants, in Europe.


Ataluren update from Genzyme

Follow-on open label clinical study announced for previous trial participants

As part of our continuing assessment, Genzyme plans to initiate a follow-on clinical study of ataluren in nmDBMD patients who previously participated in the clinical trials in Europe, Israel and Australia.
All patients who have previously participated in the clinical trials with
ataluren in nmDBMD, irrespective of their current clinical status, will be
eligible to participate.

We believe that this clinical study will allow for the collection of additional information on ataluren in nmDBMD while providing access to ataluren to all patients who have been involved in earlier clinical trials.
Currently we are working to develop the timelines and design for this clinical
study and we plan to update the community, with these additional details in the
very near future. We are reaching out to the original trial investigators to
make them aware of this new development and they may be in touch with eligible
families directly as well.

The whole article posted May 2nd 2011:


When/if Ataluren is approved by the FDA in the USA, is this an approval for all Stop-codon-boys in the USA ? Also those who didn’t participate in the previous clinical trials ?

When/if Ataluren is approvad by the European regulatory authorities, is this an approval for all Stop-codon-boys in Europe, also those who didn’t participate in the previous clinical trials ?


Can someone please help.



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Sharon or Pat

 Can the boys who were NOT in the PTC trial/Ataluren request access under the compassionate use ? 


Hi Sophie--not right now. 



Someone was asking if pulmonary function is being looked at. The answer is NO. Nothing is being looked at that was in the original trial. We do a blood draw, take height and weight and that is about all there is to it. This "extended safety study" was supposed to go thru September 2011, but we are still here in June 2012 and no one seems to know what the status is or where it is going. Don't know where the holdup is - FDA, PTC?


Also, whatever happened to the elusive muscle biopsies? There has never been anything released about those results.

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