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CureDuchenne Ventures will give Exonics Therapeutics $5 million to advance research on a gene editing technology known as CRISPR/Cas9 that is aimed at correcting most of the mutations leading to Duchenne muscular dystrophy (DMD). DMD is a rare X-linked genetic progressive muscle disease affecting nearly 15,000 boys in the United…

Thirty-three years ago, doctors told Pat Furlong that her two sons, Christopher and Patrick, had Duchenne muscular dystrophy (DMD). Ten years later, the Ohio nurse — along with other fathers and mothers of Duchenne boys — founded Parent Project Muscular Dystrophy (PPMD) to raise money for Duchenne research and ultimately to…

Santhera Pharmaceuticals is recruiting patients for a new Phase 3 clinical trial evaluating its lead candidate Raxone (idebenone) in slowing respiratory decline in Duchenne muscular dystrophy (DMD) patients being treated with glucocorticoids. Raxone is an experimental drug designed to improve energy production in the mitochondria (the cell’s powerhouse) of muscle cells. In…

Researchers have developed a new method that measures the effectiveness of a cleaning process that rids defective RNA from cells. But in muscular dystrophy (MD), an imperfect RNA is better than none at all in the production of protein, which is needed for muscle function. Now researchers say they have identified a…

Spending more time outdoors, engaging in daily activities and living in a community that accepts patients and their families all tend to improve the lives of boys with Duchenne muscular dystrophy (DMD), a European analysis finds. The analysis,“Predictors of Health-Related Quality of Life in boys with Duchenne muscular dystrophy from six European…

The current incentives offered by the U.S. Food and Drug Administration (FDA) are not efficiently stimulating orphan drug development for rare conditions like muscular dystrophy and may be benefitting pharmaceutical companies more than patients, suggest a study published in the Orphanet Journal of Rare Diseases. This is evidenced by the unmet needs of patients with such diseases and the high price of newly approved orphan drugs.

Through in-depth interviews, adult patients with Duchenne muscular dystrophy (DMD) reveal their experiences and difficulties from the point of diagnosis, through disease progression, and how their attitudes changed over time. Transitioning to a wheelchair or a respirator were identified as triggers of anxiety and worry, and Duchenne patients at…

Apabetalone (RVX-208) may become a promising therapy to treat facioscapulohumeral muscular dystrophy (FSHD), according to results announced by the pharmaceutical company Resverlogix. Researchers found this drug acts by switching off the DUX4 gene, which is abnormally active in the muscles of FSHD patients, causing inflammation and muscle atrophy. Apabetalone…

The commercialization of the recently approved drug EMFLAZA™ (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD) has been paused to reconsider the pricing of the drug.

Just days after Emflaza (deflazacort) became the first corticosteroid approved in the U.S. to treat Duchenne muscular dystrophy (DMD) regardless of mutation, the drug’s developer, Marathon Pharmaceuticals, announced it is “temporarily pausing” its sale in the U.S. to address concerns among the DMD community, particularly about the therapy’s high list price…