News

Administration of Inspra (eplerenone) to young boys with Duchenne muscular dystrophy (DMD) improves their heart function and stabilizes it in older boys, a recent study shows. The study “Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: results of a two-year open-label extension trial” was published in the Orphanet…

The U.S. Food and Drug Administration (FDA) acknowledged the filing over protest of PTC Therapeutics’ new drug application (NDA) for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The FDA granted Translarna standard review and assigned a target date of Oct. 24, 2017…

The natural sugar fructose, commonly found in fruits and vegetables, may improve a type of treatment for Duchenne muscular dystrophy (DMD), according to researchers. The study, “Fructose Promotes Uptake and Activity of Oligonucleotides With Different Chemistries in a Context-dependent Manner in mdx Mice,” was published in the journal Molecular…

Tens of thousands of businesses across the United States are taking part in this year’s 35th annual Muscular Dystrophy Association (MDA) Shamrocks campaign, which aims to raise funds for research and support programs to help people with muscular dystrophy. The MDA Shamrocks campaign is the country’s largest St. Patrick’s Day fundraising…

ATyr Pharma’s Resolaris has received orphan drug status in the United States for the treatment of limb girdle muscular dystrophy (LGMD). The Food and Drug Administration designation gives companies financial and other incentives to develop therapies for diagnosing or treating rare diseases. In the United States that means fewer…

Adults with Duchenne muscular dystrophy (DMD) may have less ability to sequentially process auditory and visual information, according to a new study. The study, “Profile of cognitive function in adults with Duchenne muscular dystrophy,” appeared in the journal Brain & Development. Most studies analyzing cognitive function in DMD focus…

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…