PPMD's Blog – June 2015 Archive (5)

FDA Accepts BioMarin's NDA for Drisapersen; Sarepta Completes NDA for Eteplirsen

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…


Added by PPMD on June 29, 2015 at 10:34am — No Comments

Webinar Recording: PF-06252616 (Anti-Myostatin)

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…


Added by PPMD on June 25, 2015 at 3:30pm — No Comments

PPMD's 2015 Connect Conference: Thank You to Our Sponsors!

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.…


Added by PPMD on June 11, 2015 at 10:30am — No Comments

PPMD's 2015 Connect Conference: Kids Track - Mad Science & Sib Shop Program

One of our most exciting and fast-growing programs at PPMD's Connect Conference is our Kids Track! This year, we will be expanding our…


Added by PPMD on June 4, 2015 at 12:30pm — No Comments

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