News

Parent Project Muscular Dystrophy (PPMD), one of the largest nonprofit organizations in the U.S. working to find a cure for Duchenne muscular dystrophy (DMD), recently announced that it will continue to support a grant for the Certified Duchenne Care Center (CDCC) Program. PPMD invests in research, advocacy, and initiatives to…

Capricor Therapeutics presented positive six-month results from the HOPE trial of CAP-1002, a stem cell-based therapy for Duchenne muscular dystrophy (DMD), at the 22nd International Congress of the World Muscle Society, Oct. 3-7 in St. Malo, France. The poster is available on Capricor’s website. The Phase 1/2 HOPE trial…

The drug discovery and development biopharma Summit Therapeutics is giving several presentations related to the ongoing PhaseOut DMD clinical trial at the 22nd International Congress of the World Muscle Society, Oct. 3-7 in St. Malo, France. One of the poster presentations includes baseline…

Positive results were reported by Catabasis Pharmaceuticals from the open-label extension MoveDMD clinical trial evaluating edasalonexent (CAT-1004) in boys with Duchenne muscular dystrophy (DMD). A global Phase 3 trial of the investigative drug is projected for the first half of 2018. The new data were presented at…

With one exon-skipping therapy — Exondys 51 (eteplirsen) — approved for Duchenne muscular dystrophy (DMD) and others in clinical trials, it’s easy to envision Sarepta Therapeutics as the “exon-skipping company.” But the idea of current exon-skipping treatments as the ultimate for DMD couldn’t be further from what Sarepta…

Parents of boys with Duchenne muscular dystrophy (DMD) as well as patient advocacy groups are praising the recent approval of Sarepta Therapeutics‘ Exondys 51 (eteplirsen) to treat the disease. But the decision by the U.S. Food and Drug Administration (FDA) to approve a treatment without clear data supporting…

A U.S. Food and Drug Administration (FDA) advisory board concluded that evidence supporting the effectiveness of Translarna (ataluren), by PTC Therapeutics, as a treatment for Duchenne muscular dystrophy is inconclusive. The opinion, which was endorsed by 10 of the 11 advisory board members at its Sept. 28 meeting, also stated that…

The newly founded Collaborative Trajectory Analysis Project (cTAP) coalition of stakeholders has invited Summit Therapeutics, a company specialized in the discovery and development of drugs targeting Duchenne muscular dystrophy (DMD), to support the development of new therapeutic products aiming to treat DMD, such as utrophin modulators. cTAP…

The Muscular Dystrophy Association (MDA) has launched its 2017 “Live Unlimited” awareness and fundraising campaign. Running until Oct. 11, it targets families affected by muscular dystrophy (MD) and other neuromuscular diseases, and aims to help patients pursue their personal and professional goals. This year’s campaign features inspiring examples:…

The nonprofit group CureDuchenne won $50,000 at a recent Shark Tank-like competition in Irvine, California, to pitch scientific projects. The “RARE Battle of the Brains” competition, held Sept. 14, was organized by Scientist.com, a marketplace for scientific services, and Global Genes, a rare disease patient advocacy group. CureDuchenne won the…