News

Findings from a recent case report published online in the journal Respiratory Medicine Case Reports suggest that patients with Facioscapulohumeral muscular dystrophy (FSHD) with nocturnal hypoventilation should have their diaphragm assessed, because nocturnal hypoventilation can be treated effectively by non-invasive ventilation. Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal…

Israeli biotech company BioBlast Pharma Ltd. has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application and is now authorized to advance development of lead product Cabaletta. The chemical chaperone is expected to become a therapeutic option for the…

The Muscular Dystrophy Association’s president and CEO opened its 2015 Scientific Conference in Washington this week by underscoring the organization’s main focus to accelerate discoveries of Duchenne muscular dystrophy (DMD) treatments for the families they serve. Steven M. Derks stated in his keynote address that the MDA has an audacious five-year plan for…

Real estate franchiser ERA is launching the ERA MDA Summer Camp Challenge, a campaign in collaboration with the Muscular Dystrophy Association (MDA) that will gather support from brokers and agents to help 1,000 children who suffer from muscular dystrophy. This year marks the fundraiser’s third year. The challenge, announced…

Researchers at The University of Chicago recently revealed that the abdominal muscles can reflect the cardiopulmonary pathology status in muscular dystrophy mouse models. The study was published in the Journal of Neuromuscular Diseases and is entitled “Cardiac function in muscular dystrophy associates…

The Stanford Comprehensive Neuromuscular Clinic at Lucile Packard Children’s Hospital (Stanford) was recently named by the Parent Project Muscular Dystrophy (PPMD) as a Certified Duchenne Care Center. The PPMD is a nonprofit organization on the forefront of the fight against Duchenne…

Biopharmaceutical company PTC Therapeutics, Inc. is not only developing a protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD), but will also expand the drug’s current developmental trial to include siblings of the patients already enrolled in their open label PTC clinical trials. Translarna will be provided…

A late-stage clinical trial performed by researchers at the University of Florida Health (UF Health) led to a new promising treatment for children with the most common form of muscular dystrophy, Duchenne Muscular Dystrophy. This condition causes the loss of mobility when the patients reach 12 years old, and…

The FSH Society, a charity organization based in Massachusetts supporting research and advocacy related to facioscapulohumeral muscular dystrophy (FSHD) has recently announced the launch of the #CureFSHD campaign. The aim of this campaign is to boost awareness and to educate people about FSHD, which is a type of…

Charles Gersbach, Muscular Dystrophy Association research grantee and Biomedical Engineering Professor at Duke University, has recently announced an enormous and potentially game-changing development in gene modification for young men and boys suffering from Duchenne muscular dystrophy (DMD). The results were published in Nature Communications in a…