Marisa Wexler, MS, senior science writer —

Real-world and natural history data could be used to supplement, or perhaps replace, the use of a placebo in future clinical trials in people with Duchenne muscular dystrophy (DMD), a new study suggests. The study, “Suitability of external controls for drug evaluation in Duchenne muscular dystrophy,”…

An imbalance in cells producing proteins called aldehyde dehydrogenases (ALDHs) — favoring fat formation in tissue — is evident in the muscles of people with Duchenne muscular dystrophy (DMD), a study reports. A better understanding of these cells could open new avenues of treatment, its researchers said. The…

An application requesting the approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping has been accepted and placed under priority review by the U.S. Food and Drug Administration (FDA), Sarepta Therapeutics announced. A regulatory decision is expected no later than Feb. 25, 2021. The…

The LELANTOS Phase 3 clinical trial, which is evaluating FibroGen‘s pamrevlumab in people with Duchenne muscular dystrophy (DMD) who are no longer able to walk, has enrolled a first patient. This global study is currently recruiting up to 90 boys and men, ages 12 and older, using…

The importance of communication and self-care for people with muscular dystrophy and other neuromuscular diseases who are pursuing their education during the COVID-19 pandemic was highlighted in a Facebook Live event hosted by the Muscular Dystrophy Association (MDA). The event featured…

An investigational gene therapy for Duchenne muscular dystrophy (DMD), called SRP-9001 micro-dystrophin, was given fast track designation by the U.S. Food and Drug Administration (FDA) its developer, Sarepta Therapeutics, announced in a press release. Fast track status is given to therapies with the potential to…

Oral Puldysa (idebenone), Santhera Pharmaceuticals‘ investigational treatment for breathing problems caused by Duchenne muscular dystrophy (DMD), will be available to eligible patients in the U.K. for another year via the renewal of its Early Access to Medicines Scheme (EAMS), the company announced. In agreeing to this renewal,…

Santhera Pharmaceuticals announced the signing of two agreements with Rutgers University related to the development of potential gene therapies for LAMA2-deficient congenital muscular dystrophy (LAMA2 MD). Included are a license agreement as well as a collaboration with Peter Yurchenco, MD, PhD, a professor at Rutgers’ Robert Wood Johnson Medical…

Both PTC Therapeutics and Sarepta Therapeutics expect to continue providing an uninterrupted supply of their Duchenne muscular dystrophy (DMD) therapies during the COVID-19 pandemic, representatives from the companies said in a Wednesday webinar hosted by Parent Project Muscular Dystrophy (PPMD). PTC is the maker of Emflaza…

The Muscular Dystrophy Association (MDA) has formed a Medical Advisory Team to provide guidance related to research and clinical care in muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular disorders. The team consists of three physicians, all experts in these conditions and leading clinicians at an…