Sarepta Therapeutics announced that the U.S Food and Drug Administration (FDA) has accepted its application for a priority review of golodirsen (SRP-4053), a potential treatment for  Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.  A decision is expected on or around Aug. 19. Golodirsen is being tested…
The U.S. Food and Drug Administration (FDA) selected  Wave Life Sciences‘ Phase 2/3 trial assessing the efficacy and safety of WVE-210201 (suvodirsen) in Duchenne muscular dystrophy for its pilot program for complex innovative trial designs. This initiative aims to modernize the design of clinical trials and advance drug development. Wave will…
The U.S. Food and Drug Administration has granted orphan drug designation to Myonexus Therapeutics‘ MYO-102, a gene therapy for limb-girdle muscular dystrophy type 2D (LGMD 2D), the company announced. The FDA grants this designation to treatments with the potential to significantly improve the life of…
Body mass index (BMI) and blood pressure are modifiable factors that could help to slow heart deterioration in patients with Duchenne muscular dystrophy (DMD), according to a retrospective study. The study, “Increased Blood Pressure and Body Mass Index as Potential Modifiable Factors in The Progression of Myocardial Dysfunction…
Metformin, a diabetes treatment, eased disease progression and improved muscle function in female — and, to a lesser extent, in male — mice altered to have congenital muscular dystrophy with laminin α2 chain-deficiency (LAMA2-CMD), a study reports. Its researchers also reported that this type of congenital muscular dystrophy — the second…
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