Patricia Inácio, PhD,  science writer—

GPM Investments, the sixth largest convenience store chain in the U.S., is running its “Tis the Season for Giving” holiday campaign throughout December to support the Muscular Dystrophy Association (MDA). Since 2012, the company’s partnership with the association has generated more than $1.2 million, which has been used…

Loss of MICU1, a key regulator of calcium balance in cells, was seen to affect muscle function and repair in a study in mice and cells from people lacking this mitochondrial protein. These findings support the involvement of MICU1 in neuromuscular disorders, its researchers said, and its potential as a…

Treatment with a cell therapy candidate called dystrophin expressing chimeras (DECs) increased dystrophin levels in heart muscle and improved cardiac function in mice with Duchenne muscular dystrophy (DMD), a study has found. This cell therapy, by Dystrogen Therapeutics, will be tested in a clinical trial involving DMD patients,…

New nine-month data from a Phase 1/2 trial show that a single administration of the gene therapy SRP-9003 (formerly, MYO-101) at low dose significantly improved functional measures and lowered the levels of a biomarker of muscle damage in three children with limb girdle muscular dystrophy (LGMD) type 2E. The trial (…

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…

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…

The U.S Food and Drug Administration (FDA) has rejected Sarepta Therapeutic’s application seeking accelerated approval of golodirsen (SRP-4053) for Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping. Golodirsen (SRP-4053) was placed under priority review by the FDA in February 2019. The FDA’s complete response…

Treatment with Exondys 51 (eteplirsen) significantly slows respiratory decline in boys with Duchenne muscular dystrophy (DMD), including those with more advanced disease, compared to standard glucocorticoid therapy, an analysis of clinical trials shows. The study “Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy”…

Santhera Pharmaceuticals has submitted a marketing authorization request to the European Medicines Agency (EMA) for Puldysa (idebenone) as a treatment for respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD). The request is made as a conditional marketing authorization (CMA), which may be granted to medicines whose benefit…