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 of the benefits, side effects, or lack of side effects?

 

Please help some of us parents who are eagerly waiting some insight.

 

Thank you.

 

Bob

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Replies to This Discussion

Hi Bob!!   My boy also has a stop codon mutation but we are not in the trial as only recently diagnosed.  Do you give your son Idebenone??  Studies show Idebenone helps the cardiac and respiratory muscles of all boys whatever the mutaton and we are planning to start Leo on this medication soon.  Regards Ruth

 

Bob,

My son Jacob has been on the PTC trial for Ataluren.

I am not sure we have seen any huge improvements. It seems there is some dystrophin production when on it.

However, I am not sure we have seen any dramatic chnages in Jacob.

At the same time when PTC/FDA discontinued the trial for the 1 year period we also felt like there was a negative change in Jacob's strength.  But this is tough to weigh against the variables because Jacob was 11 at the time and he has been slowly losing mobility at the same time.  His weakening has been slower than I thought.  He is now 12 and still some what mobile. He uses a manual wheelchair at school and we have ordered his powerchair. But he continues to walk but no longer can climb stair. I know this may not be a much help but at least you can see what impact Ataluren has done for Jacob.

Ruth,

Our son Kyle is 7.   We dropped CoQ10 and moved to Idebenone - 450mg/day (150mg x 3 times per day). I'm not sure we can prove it but very subjectively Kyle seemed to perform cognitively better with Idebenone than CoQ10.  Now, we're not sure whether it was do to him getting older or if Idebenone had any effect at all but he seems more alert and willing to take on more complex tasks without prodding.

We always, always, always try to buy everything via PPMD/Amazon via www.ShopForOurSons.com but in this case we order directly from Kirkman labs... http://www.kirkmanlabs.com.

All the best,

Bob

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Ruth Le Gal said:

Hi Bob!!   My boy also has a stop codon mutation but we are not in the trial as only recently diagnosed.  Do you give your son Idebenone??  Studies show Idebenone helps the cardiac and respiratory muscles of all boys whatever the mutaton and we are planning to start Leo on this medication soon.  Regards Ruth

 

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