Added by PPMD on January 31, 2017 at 4:08pm — No Comments
Yale New Haven Children's Hospital's Muscular Dystrophy Program became the 13th clinic named a Certified Duchenne…
Added by PPMD on January 19, 2017 at 11:30am — No Comments
Ensuring that the worlds of teens and adults with Duchenne 'grow bigger', as they grow older by expanding their reach – both literally and figuratively.
Combing your hair. Lifting a glass to your mouth. Washing your hands. Pressing elevator buttons. Zipping a coat. Opening a door. Turning on a light.…Continue
As you know, the FDA is currently reviewing Marathon Pharmaceutical’s New Drug Application (NDA) for deflazacort for the treatment of Duchenne. It is our understanding that a decision is expected to be made…Continue
Added by PPMD on January 16, 2017 at 6:30pm — No Comments
This weekend, Akashi Therapeutics announced they are working toward having three novel, complementary compounds in the clinic in 2017 with potential to treat all Duchenne patients independent of their specific genetic mutation.
This is promising news for a…Continue
Added by PPMD on January 11, 2017 at 9:00am — No Comments
On the heels of PPMD’s announcement last week of a $2.2 million grant to Nationwide Children’s Hospital’s gene therapy study being led by Dr. Jerry Mendell and Dr. Louise Rodino-Klapac, Sarepta Therapeutics today announced their commitment to the trial through a separate research agreement with Nationwide Children’s, including an…Continue
Added by PPMD on January 10, 2017 at 10:30am — No Comments
In a corporate update released today, PTC Therapeutics announced next steps for their drug, Translarna in the U.S. According to the release, PTC plans to file the Translarna New Drug Application (NDA) for nmDMD over protest with the U.S. FDA in…Continue
Added by PPMD on January 9, 2017 at 10:30am — No Comments