News

The U.S. Food and Drug Administration (FDA) and the European Commission (EC) have granted Orphan Drug designations to Solid Biosciences’ SGT-001, a micro-dystrophin gene therapy under development for the treatment of Duchenne muscular dystrophy (DMD). Clinical trials should commence in 2017, the company announced…

Consumption of large doses of a vitamin called nicotinamide riboside may help treat Duchenne muscular dystrophy (DMD) by reducing muscle inflammation, according to research in animal models of the disease by scientists at the Ecole Polytechnique Fédérale de Lausanne, Switzerland. The study, “NAD Repletion Improves Muscle Function In Muscular Dystrophy…

Duchenne muscular dystrophy (DMD) patients may have to be more alert about their chances of life-threatening constipation and they should monitor their intestinal transport vigilantly as they get older because they appear to have markedly disturbed gastrointestinal motor functions, according to a recent study. Gastrointestinal problems are not well-studied in…

The Muscular Dystrophy Association (MDA) and RYR-1 Foundation have announced an agreement to advance research and clinical care, raise awareness and improve patient, medical and public education about RYR1-related myopathies. The partnership aims to enhance the quality, quantity and scope of basic, translational and clinical research, bolster…

Researchers at the University of Missouri and their colleagues at Carnegie Institution for Science recently identified the pathway of a key gene that regulates muscle fiber production, influences muscle stem cell pool size, and leads to accelerated muscle repair. The study, “Myofiber-specific TEAD1 overexpression drives satellite cell hyperplasia and counters pathological…

The Muscular Dystrophy Association (MDA) will partner with the creative agency 50,000feet for the fifth year to help the nonprofit raise money for new research and therapies for neuromuscular diseases. The MDA will host a 2016 Muscle Team fundraiser Oct. 21 in Chicago, and 50,000feet’s brand expertise will be crucial to advance the…

The nonprofit Parent Project Muscular Dystrophy (PPMD) has awarded $239,000 to Dr. Terence Partridge, with the Research Center for Genetic Medicine at Children’s National Medical Center. The grant will support ongoing work aimed at a better understanding of exon skipping therapies for Duchenne muscular dystrophy (DMD), and their validation in future clinical trials. DMD is caused by mutations in…

Pain is a common and frequent problem among patients with Duchenne muscular dystrophy (DMD) and is associated with physical limitations and poor quality of life in these patients, according to a new study conducted by researchers in Brazil. The authors argue that knowing several aspects about pain, such as frequency,…

Resolaris, a potential intravenous protein therapy developed by aTyr Pharma for the treatment of rare myopathies is generally safe and well tolerated in patients with Facioscapulohumeral Muscular Dystrophy (FSHD) according to results presented at the 21st International Congress of the World Muscle Society.

A gene called CD40, which is an important modulator of the immune system, may determine the severity of Duchenne muscular dystrophy (DMD) according to a study published in The American Journal of Human Genetics. This finding suggests that drugs targeting CD40 may improve the symptoms of DMD.