News

BioBlast Pharma Ltd. has announced positive results from its Phase 2 open label clinical trial, HOPEMD, of Cabaletta (IV trehalose) in 25 patients suffering from oculopharyngeal muscular dystrophy (OPMD). OPMD is an uncommon disease in which progressive muscle-wasting occurs. People with OPMD have severe problems with swallowing (dysphagia), which can cause them…

Researchers at the University of Missouri have reported the complete and successful treatment of Duchenne muscular dystrophy (DMD) in dogs through injection of a viral vector carrying a dystrophin micro gene, leading to recovery of all muscles. Clinical trials involving humans are being planned as a next step for the team.

Parent Project Muscular Dystrophy (PPMD) announced the submission of results from its recent patient-centered benefit-risk assessment (PCBR) study to the U.S. Food & Drug Administration (FDA). PCBR studies incorporate patient assessment information and preferences in industry and regulatory decision-making. The PPMD study, developed in partnership with and sponsored by Santhera Pharmaceuticals, incorporated…

A heart rate-corrected 6-minute walk test (6MWT) is a more accurate outcome measure than the standard 6MWT, and should be pondered as a future outcome measure of walking capability in patients with neuromuscular diseases, according to the results of a recent study published in the journal PLOS One.

Developer of novel RNA-targeted therapeutics, Sarepta Therapeutics, recently announced the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Drugs Advisory Committee has given a tentative schedule after its review of the company’s filed New Drug Application (NDA) for the Duchenne muscular dystrophy (DMD) product eteplirsen. The committee initially set…

PTC Therapeutics, Inc., reported last week its findings from a Phase III, double-blind, placebo-controlled, 48-week ACT study of Translarna (ataluren) in patients with Duchenne muscular dystrophy (DMD). Translarna is a first-in-class, oral formulation of a protein restoration treatment for nonsense mutation DMD (nmDMD). According to the company’s report, the drug was…

In a recent study published in the journal PLOS Medicine, a team of researchers from the University of Portsmouth, U.K., led by Professor Darek Gorecki, was able to block a protein called P2RX7 which is involved in Duchenne muscular dystrophy (DMD). The results established that a specific treatment…

New research has shown that a RNA technique called “exon skipping” can successfully generate stable proteins and slow down a severe disease form of muscular dystrophy, Limb Girdle Muscular Dystrophy Type 2C. The research paper, entitled “Reengineering a transmembrane protein to treat muscular dystrophy using exon skipping”, was published in…

Alorica, an Irvine, California-based, company, recently announced it will join forces with non-profit health organization Muscular Dystrophy Association (MDA) to help fundraise for Duchenne muscular dystrophy (DMD) and the MDA. As such, Alorica will be present at the association’s annual lock-up charity event, called MDA Lock-Up, which is taking place on Oct. 15. Alorica…

The U.S. non-profit, CureDuchenne, is now making the difference in Nepal, with the organization working for about a year on site in collaboration with the Muscular Dystrophy Organization Nepal (MDON). The physical and emotional burden of Duchenne Muscular Dystrophy is experienced by patients worldwide, despite their location. Being aware of…