News

The journal Orphanet Journal of Rare Diseases has published a review highlighting the prevalence of Duchenne muscular dystrophy (DMD) as well as the disease’s costs and treatment patterns in North America and Europe.   The analysis, “The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence…

CAP-1002, developed by Capricor Therapeutics, safely and effectively treats cardiac anomalies associated with Duchenne muscular dystrophy (DMD). So says a clinical study whose preliminary results formed the basis of an April 28 webinar jointly hosted by Capricor and the nonprofit groups Coalition Duchenne and Parent Project Muscular Dystrophy. DMD, which occurs in…

Resolaris, a protein therapy developed for rare muscle diseases by aTyr Pharma, improved muscle strength and quality of life in a small study of patients with early onset facioscapulohumeral muscular dystrophy (FSHD). These findings, as well as others regarding the effect of Resolaris in patients with limb girdle muscular…

Japan has put Duchenne muscular dystrophy (DMD) drug candidate DS-5141b on its SAKIGAKE list, a designation to the U.S. Food and Drug Administration’s Fast Track Designation. United States’ fast tract. Daiichi Sankyo, the drug’s developer, announced the decision in a press release. An open-label Phase 1/2 clinical trial (NCT02667483), currently recruiting participants,…

The Duchenne muscular dystrophy (DMD) treatment Emflaza (deflazacort) was successfully acquired by PTC Therapeutics under terms agreed to in March, and may soon become available to patients at, potentially, a new and lower listing price. The now-completed agreement followed U.S. Food and Drug Administration (FDA) approval of Emflaza to treat all…

RNA sequencing may help to diagnose rare genetic muscle diseases — including muscular dystrophies — in people, according to the results of an international collaboration between several scientists. The study, “Improving genetic diagnosis in Mendelian disease with transcriptome sequencing,” was published in the journal Science Translational Medicine. Several muscle…

Researchers have found that Duchenne muscular dystrophy (DMD) patients can safely continue oral feeding by using manual assistance for coughing or mechanical insufflation-exsufflation. The study, “Rate of oral intake and effects of mechanical insufflation-exsufflation on pulmonary complications in patients with Duchenne muscular dystrophy,” was published in the Journal of…

Researchers from the National Institutes of Health have gained valuable insight into how cells in the heart and other muscles protect themselves from sudden plunges in energy production. Their findings shed light on how energy shortages affect patients with muscular disorders such as muscular dystrophy. And they may pave…

Researchers from the University of Texas Southwestern Medical Center in Dallas have corrected Duchenne muscular dystrophy (DMD) mutations in human cells and experimental animal models using a new genome editing method called CRISPR-Cpf1. These new findings offer hope in permanently eliminating genetic mutations and rescue abnormalities linked to DMD. The study, “…

New York-based Bristol-Myers Squibb announced two separate agreements with Biogen of Cambridge, Mass., and Switzerland’s Roche to license compounds that aim to treat progressive supranuclear palsy (PSP) and Duchenne muscular dystrophy (DMD). Tau protein contributes to the stability of basic cellular structures known as microtubules to transport molecules. Abnormal addition of…