Ataluren

I'm so excited about all the news about Exon 51.  It got me thinking that, oh yeah...  we're still in a trial too.  Ataluren.  What's up with that?  Is it time to get off the ataluren?  Do we just keep plugging along?  Would love to hear thoughts of others, families and providers.

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Permalink Reply by Neil McLaughlin on October 4, 2012 at 10:08am
Hi, i have heard no results from the Ataluren trials phase 3. is your child on Ataluren and is there any benefit? thanks
Permalink Reply by Jill Castle on October 5, 2012 at 2:54pm

Joanne, do you feel it's helping?

I really do feel it's helping Anthony. I wouldn't feel comfortable taking him off. But I do wish I could add some utrophin uptake drug or myostain. We need a combo approach and it's hard not being able to look at other trials because of ataluren.

I think there will be a press release next week to update us on the FDA process. I believe PTC is doing the final trial FDA required for approval.

Ataluren I believe will be very benefical when you can give it to a 3 year old and keep them on it. I think it's most beneficial when there's more muscle to preserve.Just my couple cents.

Permalink Reply by Neil McLaughlin on October 5, 2012 at 6:30pm
Hi Gill, my son is only 5 years old and 18 months and recently diagnosed. Our consultant tells us that Ataluren failed its trials, this obviously makes no sense as trials continue and private investors are investing in PTC. Any info on phase 3 trials seems near impossible to get. Can anyone shed some light on this? Does it work or can the doses be adjusted to make it work.
Thanks.
Permalink Reply by Donna on October 6, 2012 at 11:09am

Hi Neil,  my son is still on Ataluren.  It's my understanding that they are also doing another phase with just boys that have NOT been on steriods - I'm not exactly sure why as some of the population of the boys may not have been on ataluren to begin with.  I know in our case, Kyle was diagnosed in June 2008 and we had to decide almost right away if we wanted to do steriods or not because they had to be on them for 6 months in order to get in the study and that phase of the study was closing. So, Kyle HAS been on and continues to be on ataluren.  He is doing VERY well; BUT i will say we are not sure if it's because he's on steriods or if it's because he's on the ataluren or if it's because he is just doing well.... 

Permalink Reply by Neil McLaughlin on October 6, 2012 at 7:02pm
Thanks Donna for your feedback. Is there any hard evidence of the benefits? Biopsy results, reduced ck levels? This is the information that is needed. Is there anybody out there whos son is on the trial and has had these tests carried out as evidence to put forward to the fda or ema? Any further feedback would be greatly appreciated.

thanks
Permalink Reply by Donna on October 8, 2012 at 10:58am

I'm not sure what those results are in our specific case but ataluren is considered to be in orphan drug status which my understanding is very close to being approved.  I don't think it's suppose to be a cure all but to buy more time, more time on their feet, more time for finding better answers.

Permalink Reply by Marks33 on October 13, 2012 at 6:12am
Donna -please can you mail me. Had a query regarding one of your messages in this trail. Thanks.
Permalink Reply by Joanne Wechsler on October 13, 2012 at 10:24am

Yeah, I'm not sure about results.  Could be Adam was older and so much fibrosis already in action.   A doc at the conference suggested that this is not the drug we are waiting for re: stop codons, but this is a step on which the next class will be built.   



Jill Castle said:

Joanne, do you feel it's helping?

I really do feel it's helping Anthony. I wouldn't feel comfortable taking him off. But I do wish I could add some utrophin uptake drug or myostain. We need a combo approach and it's hard not being able to look at other trials because of ataluren.

I think there will be a press release next week to update us on the FDA process. I believe PTC is doing the final trial FDA required for approval.

Ataluren I believe will be very benefical when you can give it to a 3 year old and keep them on it. I think it's most beneficial when there's more muscle to preserve.Just my couple cents.

Permalink Reply by Joanne Wechsler on October 13, 2012 at 10:26am

And this is the question.  Adam had been doing really well too.  He's 16 now and no longer walking.



Donna said:

Hi Neil,  my son is still on Ataluren.  It's my understanding that they are also doing another phase with just boys that have NOT been on steriods - I'm not exactly sure why as some of the population of the boys may not have been on ataluren to begin with.  I know in our case, Kyle was diagnosed in June 2008 and we had to decide almost right away if we wanted to do steriods or not because they had to be on them for 6 months in order to get in the study and that phase of the study was closing. So, Kyle HAS been on and continues to be on ataluren.  He is doing VERY well; BUT i will say we are not sure if it's because he's on steriods or if it's because he's on the ataluren or if it's because he is just doing well.... 

Permalink Reply by Ofir Arad on October 13, 2012 at 4:27pm
Does somebody knows anything about the effects of Ataluren on cognition?
Permalink Reply by Trinh Nguyen on October 15, 2012 at 12:38am

Dear Donna,

My son gets stop codon mutation. He is 8 years old now. I want to ask your son has been on Ataluren as trial. How can we approach Ataluren ? (we are living in Vietnam ; it's difficult to approach the new research).

Tks,

Trinh
 
Donna said:

Hi Neil,  my son is still on Ataluren.  It's my understanding that they are also doing another phase with just boys that have NOT been on steriods - I'm not exactly sure why as some of the population of the boys may not have been on ataluren to begin with.  I know in our case, Kyle was diagnosed in June 2008 and we had to decide almost right away if we wanted to do steriods or not because they had to be on them for 6 months in order to get in the study and that phase of the study was closing. So, Kyle HAS been on and continues to be on ataluren.  He is doing VERY well; BUT i will say we are not sure if it's because he's on steriods or if it's because he's on the ataluren or if it's because he is just doing well.... 

Permalink Reply by Cheri Gunvalson on October 21, 2012 at 5:16pm

Ofir,

Cognition is a complicated question in many ways.  Our son is 21, has been on steroids since age 7 and ataluren for some time. I believe a negative effect the long term steroids is they have adversely affected his ability to concentrate and the ataluren has helped with this and his muscles.  That said I beleive the steroids have helped him greatly in other areas and would not take him off as is PFTs and ejection fraction is still in the low normal range.  He scored very high on his ACT, college entrance exam and is now in his 3rd year of college and set to graduate next year.  Cheri
 
Ofir Arad said:

Does somebody knows anything about the effects of Ataluren on cognition?h

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