News

The European Commission has granted Orphan Drug Designation to Benitec Biopharma’s BB-301, an investigational treatment for oculopharyngeal muscular dystrophy (OPMD). The decision represents a major advance for OPMD, a rare congenital myopathy, for which the currently used therapeutic strategies involve repetitive surgical interventions that have limited effectiveness. Orphan Drug…

CureDuchenne Cares, a community-based program providing educational and outreach workshops for parents and others caring for people with Duchenne muscular dystrophy (DMD), has just announced its planned events for 2017. CureDuchenne, the non-profit DMD research support organization, pioneered the Cares program to address a perceived urgent need to educate DMD families…

A technique called electrical impedance myography (EIM) can help detect muscle deterioration in people with Duchenne muscular dystrophy (DMD), according to a study published in the scientific journal Annals of Neurology. The technique is also able to identify the therapeutic effects of corticosteroids and could therefore be a good biomarker in clinical trials.

Many patients with myotonic dystrophy type 1 (DM1) have sleep and respiratory disorders and may benefit from targeted therapy. New research has found that the causes of sleepiness in patients with DM1 are variable, but include poor sleep patterns, obstructive sleep apnea, respiratory failure, and narcolepsy. DM1 is the most common…

A combination of two treatments significantly approved muscle function in mice with Duchenne muscular dystrophy (DMD), a study shows. Since both treatments are already approved, the findings “may open up new therapeutic avenues for Duchenne muscular dystrophy and possibly other neuromuscular diseases,” the researchers said.

The European Commission (EC) has decided to renew the conditional marketing authorization for Translarna (ataluren) to treat certain nonsense mutation Duchenne muscular dystrophy (nmDMD) patients. PTC Therapeutics can now market Translarna for nmDMD patients who are 5 or older, mobile, and living in the European Union, Iceland, Liechtenstein, and Norway. The decision follows a…

Long-term therapy with daily glucocorticoid therapy can improve clinical outcomes of young patients with Duchenne muscular dystrophy (DMD) without serious side effects, according to recent research. In “Long-Term Outcome of Interdisciplinary Management of Patients with Duchenne Muscular Dystrophy Receiving Daily Glucocorticoid Treatment,” researchers performed a retrospective study with 97 DMD…

The nonprofit Duchenne muscular dystrophy (DMD) research and advocacy organization Parent Project Muscular Dystrophy (PPMD) awarded a $2.2 million grant to Jerry Mendell, MD, PhD; co-principal investigator Louise Rodino-Klapac, PhD; and Nationwide Children’s Hospital in Columbus, Ohio, where they both work. The funding will support…

The study of rare diseases is typically short-changed with research funding, and the creation and sharing of tools to fight these disorders is the mission of a new initiative launched at the University of North Carolina at Chapel Hill. The UNC Catalyst for Rare Diseases project, funded…

Researchers found notable differences in the access to appropriate care for Duchenne’s muscular dystrophy (DMD) patients in seven European countries. In a survey known as CARE-NMD, patients in DMD registries in the countries —  Bulgaria, the Czech Republic, Denmark, Germany, Hungary, Poland and the United Kingdom — were questioned as to care…