Patricia Inácio, PhD, science writer —

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…

The Institute for Clinical and Economic Review (ICER) has set up a draft document to review evidence of the effectiveness of treatments for Duchenne muscular dystrophy (DMD). The review, “Deflazacort, Eteplirsen, and Golodirsen for Duchenne Muscular Dystrophy: Effectiveness and Value,” focuses on Sarepta Therapeutics’ Exondys 51 (eteplirsen) and…

The Hippo signaling pathway — a key regulator of tissue development, and cell growth and death — does not work as it should in Duchenne muscular dystrophy and acts to prevent, rather than aid, the regeneration of muscle cells in DMD patients, a study reports. A member of this pathway, a…

Sarepta Therapeutics‘ microdystrophin gene therapy enhanced dystrophin protein expression in the muscles of four boys with Duchenne muscular dystrophy (DMD) enrolled in a Phase 1/2 trial. More importantly, the gene therapy improved the boys’ functional performance. “The encouraging results that we previously saw and reinforced in the fourth…

The U.S. Food and Drug Administration has lifted the clinical hold on a Phase 1/2 trial evaluating Sarepta Therapeutics‘ micro-dystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD), the company announced in a press release. On July 25, the FDA placed the…