On Mar. 28, Sarepta Therapeutics announced encouraging interim results from a phase 3 clinical trial that suggest the experimental drug casimersen potentially may be effective as a treatment for Duchenne muscular dystrophy (DMD). Earlier this year, the U.S. Food and Drug Administration (FDA) accepted Sarepta Therapeutics’ New Drug Application (NDA) seeking approval under Priority Review for . . .
On Mar. 5, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug designation to Acceleron Pharma’s ACE-083, a locally acting muscle agent, for treating Charcot-Marie-Tooth disease (CMT). Delivered by intramuscular injection, ACE-083 is based on the naturally occurring protein follistatin and is designed to enhance the body’s own promoters of muscle growth, specifically in . . .
On Feb. 27, Sarepta Therapeutics announced positive interim results of a phase 1/2a clinical trial for MYO-101, a gene therapy candidate developed by Myonexus Therapeutics for patients living with limb-girdle muscular dystrophy type 2E (LGMD2E), also known as beta-sarcoglycanopathy. The first three patients dosed demonstrated significant expression of the protein beta-sarcoglycan in the muscle fiber, . . .
On Feb. 25, Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation (BTD) to AT-GAA, Amicus’ investigational combination drug therapy ATB200/AT2221 for treating late-onset Pompe disease. The company’s combination drug therapy pairs ATB200, a synthetic human acid alpha-glucosidase enzyme meant to replace the missing enzyme in Pompe disease, with pharmacological chaperone AT2221, . . .
On Feb. 14, Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for golodirsen (SRP-4053), its drug candidate for treating Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority Review status requires the FDA to review the application and . . .
On Mar. 1, the Charcot-Marie-Tooth Association (CMTA) and the Muscular Dystrophy Association (MDA) announced that they have jointly awarded a research grant totaling $276,430 over three years to Kleopas Kleopa, M.D., professor and senior consulting neurologist at the Cyprus Institute of Neurology and Genetics, Cyprus School of Molecular Medicine, in Nicosia, Cyprus. Dr. Kleopa is . . .
Results of an extension study following an MDA-supported clinical trial showed that surgical removal of the thymus in patients with myasthenia gravis (MG) offered benefits for as long as five years after surgery. The original clinical trial, which was extended for two years, showed patients who had the thymus removed had reduced muscle weakness and . . .
On Feb. 7, Cambridge, Mass.-based Solid Biosciences announced preliminary results from its phase 1/2 clinical trial, called IGNITE DMD, which is designed to assess the safety and efficacy of its lead drug candidate, SGT-001, in children and adolescents with Duchenne muscular dystrophy (DMD). DMD is caused by a mutation in the dystrophin gene on the X . . .
On Jan. 4, Italian researchers published positive results in the journal SAGE Open Medicine of a Phase 2b clinical trial (MSK-001) evaluating Firdapse (amifampridine phosphate) for treating MuSK antibody positive myasthenia gravis (MuSK-MG). Their results showed the drug to be safe and effective in treating patients with this rare disease. The study was partially funded . . .
On Jan. 4, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to Tacoma, Wash.-based Revalesio Corporation’s experimental drug for amyotrophic lateral sclerosis (ALS), RNS60. Fast Track status helps to shorten the time it takes to develop and approve a drug that’s been designed to treat a serious condition and fill an unmet . . .