Premature Stop Codon


Premature Stop Codon

Members: 76
Latest Activity: Jul 5, 2016


Ataluren - PTC124

Started by Chad Studebaker. Last reply by John Smith Dec 13, 2010. 3 Replies

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Comment by Teresa Wood on November 7, 2009 at 10:12pm
My son is 7 and has a premature stop codon. I am excited about the PTC 124 release. But like everyone else it can't come a moment too soon.
Comment by Cheri Gunvalson on September 19, 2009 at 11:12am
There is plans for a nonambulatory PTC 124 (ataluren) in 2010. As you all know the drug is scheduled to be approved in 2010. The only reason they would be planning a trial for the nonambulatory is that it will be approved only for the ambulatory which is sad as muscle is muscle, if it helps the arm muscles of the ambulatory boys it will help the nonambulatory. This will have adverse effects for the nonambulatory for future drugs as it is not cost effective to do trials on the nonambulatory. This is very sad for the older boys. Many drug companies get around this by releasing the drug to the FDA for expanded access but PTC is unwilling to do so. Cheri, mom of Jacob 18 yrs old with a premature stop on exon 5
Comment by Perlita & Gordy Hains on September 15, 2009 at 12:24am
yes, premature stop codon is the same as point mutation, sometimes called Nonsense mutation. At conference they mentioned that the drugs are working well & that they are trying to "fast track" it so the boysthat have stop codons can get it. Not sure about Phase 3 trials, what they did say was that once this part is over it will take 6 months to compile data, then an additional 6-12 mths for the FDA to approve. The FDA apparently likes what going on so there should not be many, if any hold ups. My Levi is 3, so by the time he's 4-5 PTC124 (aka Ataluren) BETTER be out!!! we can't wait. There are 3 upcoming Utrophin trials coming SOON, Italy, US & ?... They say Utrophin will help ALL DMD boys... lots of positive coming! keep in touch your friend perlita
Comment by Tracey Hartz on September 13, 2009 at 8:03pm
Hi-just found out that my son Seth has a premature stop codon (same as a point mutation, right?) I know he can't get into the current trial, but how far out is FDA approval?? They still need to do a phase III trial, right? And if so, do the participants have to have been in the previous phases? I'm not quite sure how it all works. Thanks!
Comment by Perlita & Gordy Hains on April 24, 2009 at 8:34pm
New to this Premature stop codon world - all I know is that WHEN PTC124 becomes FDA approved he will get! That's the only bright spot in this nightmare.
Comment by Joanne Wechlser on September 14, 2008 at 9:46am
Hi there,
If anyone is in the 2a extension study, there is another group for a discussing progress with this subgroup. Would love to hear where everyone (38 boys) is at with this process. Please join 2a Group too. Joanne
Comment by Cathy on August 8, 2008 at 4:53pm
Jil, are you talking about the school nurse? If so, my grandson had the same problem. The school cannot give it to him because it is not approved. The doctors changed his dosage schedule to get around it. He takes it just before school, right after school and later at night.
Comment by Jill Castle on August 8, 2008 at 3:33pm
Let me know if anyone who is on the trial for PTC124 and is having trouble getting their nurse to administer the compound because it's not FDA approved. I have the IEP & 504 information to help.

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