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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.