The U.S. Food and Drug Administration has granted accelerated approval to Exondys 51 (eteplirsen), by Sarepta Therapeutics, making it the first drug approved to treat Duchenne muscular dystrophy (DMD) patients. Specifically, this treatment is for DMD patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping.
The U.S. Food and Drug Administration has approved Sarepta Therapeutics’ therapy Exondys 51 (eteplirsen) as a treatment for Duchenne muscular dystrophy patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. While this particular mutation affects only about 13 percent of the DMD patient population, Exondys 51 is now the…
Researchers at Stony Brook University, in New York, found that a protein (dystroglycan) of the muscle whose impaired activity contributes to the pathology of Duchenne muscular dystrophy, also plays a key role in brain development. Because previous research has shown that Duchenne muscular dystrophy (DMD) patients may also develop neurological conditions…
Australia’s drug regulatory agency, the Therapeutic Goods Administration (TGA), has granted Santhera Pharmaceuticals’ lead product Raxone (idebenone) orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). Raxone (sold in some markets under the trade name Catena) has already received a similar designation from European, Swiss, and…
PTC Therapeutics marked Sept. 7 — World Duchenne Awareness Day — by announcing the four winner of the company’s 2016 global STRIVE Awards (Strategies to Realize Innovation, Vision and Empowerment) program. STRIVE awards are granted in recognition of extraordinary excellence and innovation by nonprofit organizations that serve rare disease communities, such as…
Alysia Vrailas-Mortimer and her research team at Illinois State University have received a $435,000 grant from the National Institutes of Health (NIH) to continue unveiling the genetics of muscular dystrophy, specifically “limb girdle” muscular dystrophy. The team studies this special form of muscular dystrophy, which attacks muscles around the shoulders…
The U.S. Food and Drug Administration (FDA) has allowed an investigational new drug (IND) application for Carmeseal-MD in Duchenne muscular dystrophy (DMD), according to Phrixus Pharmaceuticals. Carmeseal-MD (Poloxamer-188 NF, or P-188 NF) is the first disease-modifying agent in diseases characterized by membrane instability such as Duchenne MD or limb…
CureDuchenne, the Duchenne muscular dystrophy nonprofit group engaged in disease advocacy and research funding, announced on Sept. 7 — World Duchenne Awareness Day 2016 — that it has received a coveted STRIVE Award in recognition of its efforts. The awards were created by PTC Therapeutics to recognize, support, and honor…
A form of vitamin B3 taken as a supplement may reverse muscle frailty associated with aging by boosting levels of NAD, a molecule that helps generate energy from food nutrients, researchers report. The findings may lead to new ways of ameliorating certain aspects of muscle dystrophy. The recent study, “Loss of NAD…
Results from a Phase 3 clinical trial comparing deflazacort and prednisone, two corticosteroid drugs under evaluation as potential treatments for Duchenne muscular dystrophy, showed that both deflazacort and prednisone improved muscle strength compared to a placebo, meeting the primary endpoint of the study. But deflazacort caused less weight gain than prednisone, according to…
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