News

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

Three new animal-model tests of a potential gene therapy for oculopharyngeal muscular dystrophy (OPMD), known as BB-301, are planned, Benitec Biopharma announced. Results are expected to support a request for a Phase 1 clinical trial in patients. OPMD results from a faulty PABPN1 gene, leading to a protein…

To help families find the ideal equipment at every stage of need for those with Duchenne muscular dystrophy (DMD), CureDuchenne launched on its website a comprehensive and interactive guide. Presenting the latest offerings together in one place, the digital Durable Medical Equipment Guide (DME) is meant to reduce frustration…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to suvodirsen as a treatment for people with Duchenne muscular dystrophy (DMD). The agency’s decision — which will expedite the review process for suvodirsen — was based on experimental and preclinical data demonstrating the treatment’s…

Due to a lack of efficacy, Acceleron Pharma is halting the clinical development of its ACE-083 candidate for the treatment of facioscapulohumeral muscular dystrophy (FSHD) . Topline results from a Phase 2 clinical study showed that ACE-083 was well-tolerated and could significantly increase muscle mass, which was…

Researchers have identified a new set of blood biomarkers of Duchenne muscular dystrophy (DMD) and of response to standard glucocorticoid therapy. The study, conducted by U.S. and Canadian researchers, also found that glucocorticoid treatment can result in significant changes in the levels of proteins not related to DMD, highlighting…

A review study highlights the benefits of adding an extra protein domain —  called neuronal nitric oxide synthase (nNOS) — to microdystrophin gene therapy. nNOS is an important player in regulating blood flow to muscle cells, and the reviewed evidence suggests it protects muscle against restricted blood flow (a condition…

Twenty-five research grants totaling more than $6.6 million have been awarded by the Muscular Dystrophy Association  (MDA) to support studies into the mechanisms of neuromuscular diseases, including Duchenne and other muscular dystrophies. The funding will allow researchers not only to investigate the causes of neuromuscular diseases, but also to…

BBP-418, an oral treatment candidate for patients with limb-girdle muscular dystrophy type 2I (LGMD2I), is being developed by ML Bio Solutions, a new subsidiary of BridgeBio Pharma, the company has announced. BridgeBio intends to start a natural history study by…

As part of an effort to heighten awareness of its work in research, treatment and care — as well as to raise funds — the Muscular Dystrophy Association (MDA) has proclaimed September MDA National Muscular Dystrophy Awareness Month. The launch dovetails with World Duchenne Awareness Day, observed annually…