Patricia Inácio, PhD, science writer —

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…

New nine-month data on four boys with Duchenne muscular dystrophy (DMD) enrolled in Study-101 testing Sarepta Therapeutics‘ micro-dystrophin gene therapy continues to show “very encouraging” results, company officials said. These updated data, given in a recent investors’ webinar, show the gene therapy resulted in 81.2% increase in dystrophin protein…

Santhera Pharmaceuticals intends to file for a conditional marketing authorization with the European Medicines Agency (EMA) for idebenone — to be marketed as Puldysa — as a treatment for respiratory dysfunction in Duchenne muscular dystrophy (DMD). As it continues to gather data on the therapy, the company…

Young boys with Duchenne muscular dystrophy (DMD) have a higher rate of bone fractures than healthy children, a retrospective study shows. The fractures are more frequent in lower limbs and decrease the patients’ ability to walk, leading to wheelchair dependence. The study, “Fractures and bone health monitoring in boys…