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The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
A gene therapy treatment may restore the genetic mutations of myotubular myopathy, a type of muscular dystrophy that affects the skeletal muscles. In a previous study, a team of researchers at the University of Washington School of…
The Muscular Dystrophy Association (MDA) has awarded $7 million in grants to 29 top researchers for projects it says will potentially make a big impact on muscular dystrophy and other life-threatening diseases. The investment reflects the nonprofit organization’s plan to double spending on drug development and clinical trials by 2020. Each year,…
The U.S. Food and Drug Administration has approved Sarepta Therapeutics’ therapy Exondys 51 (eteplirsen) as a treatment for Duchenne muscular dystrophy patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. While this particular mutation affects only about 13 percent of the DMD patient population, Exondys 51 is now the…
BioMarin Pharmaceutical Inc. has announced the withdrawal of its Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) for Kyndrisa (drisapersen), a drug developed for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The decision follows deliberations at the May 2016 Committee for Medicinal Products for…
UPDATE: Since this story was filed, Reuters news service is reporting that even more quickly than we had anticipated was likely, the expert panel convened by the FDA this week to review clinical trial data pertaining to Sarepta’s Duchenne Muscular Dystrophy (DMD) drug candidate Eteplirsen has denied…
The glucocorticoid deflazacort might shortly be approved for treatment of Duchenne muscular dystrophy (DMD) in the US. In preparation for the approval process, Marathon Pharmaceuticals have released new data showing that in young rats, the drug causes a slowing of growth but is otherwise safe. Deflazacort has been used…
Summit Therapeutics plc announced the publication of a study into new imaging techniques that appear to reliably and reproducibly measure utrophin protein levels and muscle fiber regeneration in muscle biopsies in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) patients. The study is important to the company because one of its…
Sarepta Therapeutics, Inc., announced that a U.S. Food and Drug Administration (FDA) advisory committee has been forced to postpone Friday’s scheduled hearing to review the company’s New Drug Application (NDA) for eteplirsen, a potential treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. A severe winter storm starting…
Marathon Pharmaceuticals has announced the launch of ACCESS DMD, its expanded access program (EAP) to provide deflazacort to qualified patients with Duchenne muscular dystrophy (DMD) under the authorization of the U.S. Food and Drug Administration (FDA). Marathon Pharmaceuticals, LLC, a biopharmaceutical company that develops treatments for rare neurological diseases,…
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