News

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…

Lipid-lowering medications are used by many people to reduce risk of heart disease and other medical conditions. A new study from Seattle, Washington, indicates an unexpected use of simvastatin, commonly known by the brand-name Zocor in the United States. According to the report, simvastatin may help skeletal muscles become more healthy in…

Privately-held biopharmaceutical company, Tarix Orphan LLC, has just announced the US Food and Drug Administration has granted its lead candidate for Duchenne Muscular Dystrophy, TXA127 (angiotensin 1-7), Fast Track Designation for its potential in lessening damage and fibrotic activity in skeletal muscle. Under a previously filed Investigational New Drug (IND)…