Big news in exon skipping therapy today! BioMarin announced this morning that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen.

We are such a fortunate…

On June 3, 2015, Dr. Michael Binks, Vice President for Rare Disease Clinical Research at Pfizer, presented information about the upcoming clinical trial of  PF-06252616. The webinar provides an overview of Pfizer’s approach to advancing the science of an anti-myostatin monoclonal antibody as an investigational compound for potential therapeutic use in muscle wasting diseases, such as Duchenne muscular dystrophy. Dr. Binks gives a presentation and answers submitted…

Parent Project Muscular Dystrophy is excited to name Duke Children’s Hospital & Health Center (Duke Children’s) a Certified Duchenne Care Center. Duke…

Today, we are blessed with an overwhelming number of industry and corporate partners who have committed themselves to the fight to end Duchenne. This kind of commitment was unheard of when PPMD started 21 years ago. Because of the dedication of these companies and others, we are stronger as a community. We are closer to ending Duchenne.…