Catabasis has announced top-line results for Part B of the MoveDMD® Trial for Edasalonexent (CAT-1004) in…

Robotics.

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.…

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…

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…

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…

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…