PIONEERS IN DMD THERAPY

PTC Therapeutics was one of the first pharmaceutical companies to develop a treatment for Duchenne muscular dystrophy (DMD). Developing ataluren (Translarna™), a treatment that works on a nonsense mutation in the dystrophin gene took 20 years. When PTC started its research, there was no North Star Ambulatory Assessment (NSAA), a mobility test that measures the results of treatment. The company began with a blank sheet. Its hard work and persistence encouraged other pharmaceutical companies to jump in and investigate new treatments for DMD. Continue Reading here.

GROUNDED IN THE DMD COMMUNITY

Sarepta Therapeutics, Inc., a biopharmaceutical company, is working to unlock the potential of RNA-based and gene therapy technologies for the treatment of serious and life-threatening diseases like Duchenne muscular dystrophy (DMD). Sarepta’s primary focus is to rapidly advance new treatments for DMD. Continue reading here.