The Muscular Dystrophy Association (MDA) has awarded $7 million in grants to 29 top researchers for projects it says will potentially make a big impact on muscular dystrophy and other life-threatening diseases. The investment reflects the nonprofit organization’s plan to double spending on drug development and clinical trials by 2020. Each year,…
A new article reviewing the effect of Duchenne muscular dystrophy (DMD) on heart function has been published in the Journal of Neuromuscular Diseases. The article titled “Cardiac Involvement Classification and Therapeutic Management in Patients with Duchenne Muscular Dystrophy” proposes a classification for the effect of the disease on the heart and outlines the therapeutic management techniques against heart failure.
Bone health varies greatly among patients with facioscapulohumeral muscular dystrophy (FSHD), according to the results of a small group study, and tests for strength and functionality may identify those with higher risks for decreased bone health. The study “Bone Health in Facioscapulohumeral Muscular Dystrophy: A Cross-Sectional Study” was published…
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…
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