Audentes Therapeutics Announces Plans to Develop Gene-Targeted Therapies for Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1

On April 8, Audentes Therapeutics announced that it will expand its gene-targeted therapy platform to include developing experimental drugs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The company has entered into a licensing agreement with Nationwide Children’s Hospital in Ohio and will collaborate with leading researchers in the field, Kevin M. Flanigan, . . .

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Sarepta Therapeutics Announces Positive Interim Results from Study of Casimersen to Treat DMD Amenable to Exon 45 Skipping

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 . . .

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Buy a Shamrock, Grow Hope

It’s March, and at MDA that means only one thing: It’s Shamrocks season! Each St. Patrick’s Day season for the last 37 years, MDA has partnered with 20,000 retailers nationwide to raise a little green (more than $323 million, to be exact) both to send kids to MDA Summer Camp and to fund groundbreaking research . . .

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FDA Accepts Sarepta Therapeutics’ New Drug Application (NDA) for Golodirsen to Treat DMD Amenable to Exon 53 Skipping

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 . . .

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Newborn Screening for DMD and BMD: MDA Needs Your Help

Early identification and treatment for neuromuscular disorders are essential to optimize health outcomes. Newborn screening, which identifies health issues via a blood test taken soon after birth, is essential to ensure that infants born with serious but treatable disorders have the best possible chance at receiving the care and support services they need as early . . .

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Wave Life Sciences Announces Positive Results of Phase 1 Trial of WVE-210201 for Duchenne Muscular Dystrophy

Today, Wave Life Sciences announced that its Phase 1 trial of WVE-210201, an investigational, exon skipping therapy for boys with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, showed positive safety and tolerability data. Based on these results, Wave announced that it will initiate a Phase 2/3 clinical trial of WVE-210201 in 2019. Wave . . .

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