News

While current studies in Duchenne muscular dystrophy (DMD) mainly explore gene and exon-skipping therapies, other options might exist in drugs already on the market. A study, published in the journal Rare Diseases, shows that the cholesterol-lowering drug simvastatin is as effective in improving muscle health as the best genetically based experimental…

Santhera Pharmaceuticals announced updates on regulatory filings for Raxone (idebenone), saying it plans to submit comprehensive briefing material and a meeting request to the Food and Drug Administration (FDA) to discuss filing a New Drug Application (NDA) for the treatment of Duchenne Muscular Dystrophy (DMD) patients who are not taking concomitant…

An expert panel convened by the U.S. Food and Drug Administration (FDA) recently voted against granting accelerated approval to the novel Duchenne muscular dystrophy (DMD) drug candidate, eteplirsen. The vote came after a review of clinical trial data and 11 hours of hearing testimony that focused on the drug’s efficacy,…

The Race to Yes initiative is urging the U.S. Food and Drug Administration (FDA) to carefully consider the evidence presented at the latest Advisory Committee meeting and to approve fast access for the Duchenne muscular dystrophy drug, eteplirsen. “Monday’s advisory committee meeting on eteplirsen saw a unified show of support from the…

Lowe’s, a nationwide home improvement company, announced it has raised over $7.5 million across its more than 25,000 U.S. retail stores for the Muscular Dystrophy Association’s (MDA) Shamrocks program. Lowe’s is MDA’s largest Shamrocks retailer and has been involved with the program for 15 years, donating more than $57 million…

University of Iowa researchers, working in a mouse model of muscular dystrophy (MD), have established new molecular signatures associated with membrane protein complexes that typify the disease, possibly opening the way to new and suitable therapies, according to the study, “Molecular signatures of membrane protein complexes underlying muscular dystrophy,” published in …

UPDATE: Since this story was filed, Reuters news service is reporting that even more quickly than we had anticipated was likely, the expert panel convened by the FDA this week to review clinical trial data pertaining to Sarepta’s Duchenne Muscular Dystrophy (DMD) drug candidate Eteplirsen has denied…

Current drug development approaches for muscular dystrophy have focused mainly on loss of walking ability and do little for heart dysfunction, which often lead to premature death in patients suffering Duchenne and Becker muscular dystrophies (DMD and BMD). Attempts to develop therapies targeting heart dysfunction are underway, however, with clinical trials…

Mice lacking the immunomodulatory protein osteopontin have fewer Duchenne muscular dystrophy (DMD) symptoms, a finding researchers attributed to a switch in macrophage subsets. The study supports the notion that DMD patients could benefit from treatment with osteopontin blockers. Scientists know that immune cells, recruited to damaged muscles to initiate repair, ultimately end…

Marathon Pharmaceuticals recently presented additional results from a Phase 3 clinical trial of deflazacort, an investigational glucocorticoid corticosteroid that the company hopes will be approved for the treatment of Duchenne muscular dystrophy (DMD) early next year. The data was presented at the 68th American Academy of Neurology (AAN) Annual Meeting, running through April…