News

The FSH Society, a Massachusetts-based charity supporting research into facioscapulohumeral muscular dystrophy (FSHD), has announced that it recently completed the 2nd edition of the FSHD Trial Preparedness Workshop, which was held between May 29 and 30, 2015 at the University of Rochester Medical Center in Rochester, New York. The workshop included over 50 stakeholders from around…

A new study published in the journal Human Molecular Genetics revealed a new therapeutic approach that could potentially alter the progression of muscle damage and dysfunction in patients with myotonic dystrophy. The study is entitled “TWEAK/Fn14, a pathway and novel therapeutic target in myotonic…

In a recent study published in Oncotarget, researchers found an inverse correlation between the level of muscle fibrosis, utrophin and the number of revertant myofibers in Duchenne Muscular Dystrophy (DMD). Results from this study reveal common links between the fibrotic and utrophin-synthesis pathways and offer new insights into…

A new collaborative group involving academic and industry researchers and patient advocacy groups conducted a large-scale study to find serum biomarkers for Duchenne muscular dystrophy (DMD). The study was recently published in the journal Proceedings of the National Academy of Sciences (PNAS) and is entitled “…

The Muscular Dystrophy Association (MDA) has begun another season of its well known summer camp, marking its 60th year in operation. The MDA summer camp aims to support families by hosting thousands of children struggling against muscular dystrophy and similar conditions that seriously limit their mobility and muscular strength. The summer camp assists kids in helping them…

Managing muscular dystrophy (MD) is not an easy task, and it depends not only medical intervention, but also on methods to improve patients’ quality of life. “Supporting People & Pets through Opportunity and Training” (SPPOT) is a community interest company focused on that purpose, helping to train…

Germany’s Federal Joint Committee (G-BA) published a positive Benefit Assessment for Translarna (ataluren), including an evaluation of the benefits of the drug in treating nonsense mutation Duchenne Muscular Dystrophy (nmDMD) in ambulatory patients older than five years. The therapy, which was developed by PTC Therapeutics, was rated “three” — or minor additional benefits…

Sarepta Therapeutics, Inc. , a biopharmaceutical company focused on developing innovative RNA-based therapeutics, has announced a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) pertaining to its lead product candidate eteplirsen — a Duchenne muscular dystrophy (DMD) treatment. Sarepta and the agency are agreed…

Jordan McLinn is turning 6 years old and his mom has a single birthday wish: the possibility of watching him grow up and have the opportunity of becoming the firefighter that he dreams of becoming. Jordan suffers from a deadly type of muscular dystrophy, Duchenne, which will begin to immobilize him within a few years and dramatically…

BioMarin Pharmaceutical Inc. announced the presentation of data regarding disease progression in patients with Duchenne Muscular dystrophy (DMD) at the 67th American Academy of Neurology (AAN) 2015 Annual Meeting in Washington, D.C., April 18-25. The presentation was entitled “Evaluating the Progression of Physical Impairment, Activity Limitation, and Quality…