News

Wave Life Sciences, Ltd., is planning to begin clinical testing of its next-generation nucleic acid therapy for patients with Duchenne muscular dystrophy (DMD), which the company says has shown promise in pre-clinical trials. Its initial DMD candidate skips exon 51 in the dystrophin gene. Data from early studies showed a roughly…

Researchers have discovered the molecular mechanisms responsible for the heart dysfunctions that mark myotonic dystrophy, and reported they appear to be linked to a defective processing of the cardiac sodium channel (SCN5A) RNA. Their study, published in Nature Communications, is titled “Splicing misregulation of SCN5A contributes to cardiac-conduction delay and…

Genetic data generated through thousands of experiments and freely available to access may — with the help of the Internet and a little ingenuity — change the future of how new drugs are brought to the market. According to Professor Atul Butte, a director of the University of California Institute of…

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…