Magdalena Kegel,  —

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…

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.

The first patients with Duchenne muscular dystrophy (DMD) have been enrolled into the U.K.’s Early Access to Medicines Scheme (EAMS) for Santhera Pharmaceuticals’ Raxone (idebenone), giving patients access to the treatment before its regulatory approval. Raxone was included in EAMS after the Medicines and Healthcare Products Regulatory Agency (MHRA)…

Parent Project Muscular Dystrophy (PPMD) will host a one-hour webinar at 2 p.m. Eastern Time on Wednesday, Sept. 6, that will focus on an upcoming clinical trial exploring gene therapy for Duchenne muscular dystrophy. The webinar will be led by Dr. Jerry Mendell, who, together with fellow researcher Dr. Louise…

Treadmill training using a voluntary-driven exoskeleton might be a safe approach to improve movement and exercise capacity in patients with limb-girdle muscular dystrophy, according to a small study by researchers at Ruhr University Bochum in Germany. The study, “Treadmill Training with HAL Exoskeleton — A Novel Approach for Symptomatic…

The chemical structure of a drug can make the entire difference between a success and a failure in drug development. Of two drugs that used the same approach to treat Duchenne muscular dystrophy — Exondys 51 (eteplirsen) and Kyndrisa (drisapersen) — only one became approved. Researchers from The Ohio…

The U.S. Food and Drug Administration has approved Capricor Therapeutics’ plan for further development of its CAP-1002 stem cell therapy for Duchenne muscular dystrophy, paving the way for another Phase 2 clinical trial of the treatment this year. The thumbs-up came when Capricor received official minutes from a…

Long after it arrived elsewhere, Emflaza (deflazacort) became the first corticosteroid that the U.S. Food and Drug Administration approved to treat all forms of Duchenne muscular dystrophy — in February 2017. But its U.S. introduction was far from smooth. In fact, it took three more months and a change of…

Healthcare costs for patients with Duchenne muscular dystrophy (DMD) are about 10 times those of healthy people, and increase as patients age, according to a new analysis of claims data. The figures, which may aid in healthcare planning and evaluating costs of emerging treatments, were — for the first time —…

Using glucocorticoid steroids once a week instead of once a day to slow the progression of muscular dystrophy (MD) could prevent downsides of the treatment, including accelerated muscle wasting, a study reports. Researchers at the Feinberg School of Medicine at Northwestern University discovered that mice with Duchenne muscular dystrophy fared better…