Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

I realize it is so difficult to see trials targeting specific mutations and feel like you are on the outside looking in, wondering when it will be your son’s turn. Prosensa and AVI’s trials are moving forward as well. More trials are in the planning stages – Sildenafil, Losartin, IGF-1 and others. Still some of our boys do not fit the criteria, either based on mutation or because they are not walking.

So this trial fills a hole. It is a safety trial but will validate outcome measures for the non-ambulatory boys. Outcome measures in the non-ambulatory population have been a significant barrier for inclusion in clinical trials and for this reason, trials have focused on ambulatory boys. How to measure? What to measure?
There has been considerable discussion about cardiac and pulmonary outcome measures, clinicians concerned about the length of time required to demonstrate decrease, stabilization or benefit.

While the Ataluren trial will include cardiac and pulmonary endpoints, it will include a variety of functional measures, assuming one or more will be validated as THE outcome measure(s) for the non-ambulatory population.

And then, at the end of the day, non-ambulatory boys will not feel like they are at the end of the line.

For more on this trial, click here.

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Comment by Paul Cliff on December 26, 2009 at 11:46am
Since most of the non-ambulatory patients are in their teens or later, has anyone given any thought to asking them what functionality is most important to their quality of life. I recall that in the notes from a parent conference of a couple of years ago, you mentioned how some of the adult DMD men talked about what was important to them. Has anyone communicated those desires to PTC?
Comment by Ian Anthony Griffiths on December 12, 2009 at 10:02am
Thanks Pat.
Comment by Darcy Tumminello on December 12, 2009 at 6:50am
Makes the Holidays brighter!
Comment by cheryl cliff on December 11, 2009 at 7:05pm
This is very good news! Not only for this trial but others too. Hopefully, soon PTC will consider compassionate use for those who can't wait any longer. I don't see how compassionate use could harm PTC at this point - they look pretty solid with Ataluren.

Comment by Pat Furlong on December 11, 2009 at 5:27pm
Happy Holidays to you too, Ian.
Comment by Ian Anthony Griffiths on December 11, 2009 at 4:22pm
welcome news indeed! Time is our enemy, im glad they are looking at the non-ambulatory now. Keep pressing them Pat, happy holidays.


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