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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…

Synpromics and Solid Biosciences have established a collaborative agreement to develop new potential gene therapies for the treatment of Duchenne muscular dystrophy (DMD). According to the partnership terms, Solid Biosciences will gain access to muscle-selective promoter candidates developed by Synpromics, which are…

PTC Therapeutics shared additional data from a Phase 3 trial of Translarna (ataluren), showing that the treatment slightly slowed disease progression in ambulatory boys with Duchenne muscular dystrophy. The data was presented at the recent American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting in Phoenix, Arizona.

Phrixus Pharmaceuticals and several Duchenne muscular dystrophy (DMD) organizations will jointly conduct the first open-label trial of Poloxamer-188 NF, the active ingredient in Carmeseal-MD, to treat DMD in patients who can no longer walk. The trial, to take place at Cincinnati Children’s Hospital, will evaluate the drug’s performance on respiratory…

For the ninth year in a row, “Coach to Cure MD” and Parent Project Muscular Dystrophy (PPMD) will mark Duchenne Awareness Month  by using college football games to raise awareness of Duchenne muscular dystrophy (DMD) and money to promote research into treatments and a possible cure. The effort, a partnership between the…