News

BREAKING NEWS: FDA Approves Sarepta’s Exondys 51 (Eteplirsen) as First Therapy for Duchenne MD

The U.S. Food and Drug Administration has approved Sarepta Therapeutics’ therapy Exondys 51 (eteplirsen) as a treatment for Duchenne muscular dystrophy patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. While this particular mutation affects only about 13 percent of the DMD patient population, Exondys 51 is now the…

Protein Involved in Duchenne MD Influences Brain Wiring, Study Says

Researchers at  Stony Brook University, in New York, found that a protein (dystroglycan) of the muscle whose impaired activity contributes to the pathology of Duchenne muscular dystrophy, also plays a key role in brain development. Because previous research has shown that Duchenne muscular dystrophy (DMD) patients may also develop neurological conditions…

PTC’s STRIVE Awards Honor 4 DMD Advocacy Groups on 4 Continents

 PTC Therapeutics marked Sept. 7 — World Duchenne Awareness Day — by announcing the four winner of the company’s 2016 global STRIVE Awards (Strategies to Realize Innovation, Vision and Empowerment) program. STRIVE awards are granted in recognition of extraordinary excellence and innovation by nonprofit organizations that serve rare disease communities, such as…

‘Limb Girdle’ MD Genetics Research Gets Boost from NIH Grant

Alysia Vrailas-Mortimer and her research team at Illinois State University have received a $435,000 grant from the National Institutes of Health (NIH) to continue unveiling the genetics of muscular dystrophy, specifically “limb girdle” muscular dystrophy. The team studies this special form of muscular dystrophy, which attacks muscles around the shoulders…

CureDuchenne Wins STRIVE Award to Expand Services for DMD Community

CureDuchenne, the Duchenne muscular dystrophy nonprofit  group engaged in disease advocacy and research funding,  announced on Sept. 7 — World Duchenne Awareness Day 2016 — that it has received a coveted STRIVE Award in recognition of its efforts. The awards were created by PTC Therapeutics to recognize, support, and honor…