Two new studies that can explain more than half of the variability in disease progression seen in Duchenne muscular dystrophy (DMD) have recently been published. The results have important implications for the design of new and effective clinical trials testing the efficacy of potential DMD drugs.
News
Patients with Duchenne muscular dystrophy (DMD) who carry a variant in the CD40 gene, which encodes a protein of the immune system, have loss of ambulation at an earlier age, according to a study published in the American Journal of Human Genetics. The study, “Association Study Of Exon…
Parent Project Muscular Dystrophy (PPMD) has designated the Center for Duchenne Muscular Dystrophy (CDMD) at UCLA its 12th Certified Duchenne Care Center. PPMD is a nonprofit that works to serve the Duchenne muscular dystrophy (DMD) community through researching new treatments and demanding the best care for patients. Its…
The Assistance Fund has announced the opening of a new service, called the Duchenne Muscular Dystrophy Financial Assistance Program, to help to people living with Duchenne muscular dystrophy (DMD) meet their medical expenses. The Assistance Fund is an independent charitable patient assistance foundation that helps patients and families with such expenses as co-payments and…
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…
News
Road to Better Muscular Dystrophy Treatment Possible with Discovery of New Muscle Repair Pathway
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…
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