Parent Project Muscular Dystrophy (PPMD) recently recognized Children’s Hospital Los Angeles as a Certified Duchenne Care Center (CDC). The hospital is one of only 10 facilities in the U.S. to hold this distinction and the only one in Southern California since the PPMD began qualifying healthcare centers in 2014.
Promising new biomarkers to detect structural changes in the hearts of people with Duchenne and Becker muscular dystrophy have been identified, according to a study published in the Journal of Cardiovascular Magnetic Resonance. Biomarkers, like miRNAs, that circulate in the blood and are easily detectable can help diagnose and monitor chronic heart-muscle disease, or…
MDA Southern Wisconsin, together with presenting sponsor Harley-Davidson Motor Company, will host the 23rd Annual Black-N-Blue Ball charitable gala in Milwaukee on May 20, aiming to raise $1 million for adults and children with muscular dystrophy. Jann Carl of “Entertainment Tonight” will serve as master of ceremonies. At the event,…
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…
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