News

The U.S. Food and Drug Administration has granted RASRx1902 orphan drug status to treat Duchenne muscular dystrophy (DMD), says its developer, RASRx of Newport Beach, California. The Orphan Drug status is given to therapies intended to prevent and treat rare life-threatening or chronically debilitating conditions that affect no more than five…

Simultaneously inhibiting three proteins can increase muscle mass, a finding that could help those with diseases such as muscular dystrophy and cancer that cause muscle deterioration, an Australian study reports. The study, “Specific targeting of TGF-β family ligands demonstrates distinct roles in the regulation of muscle mass in health and…

On June 20, the Massachusetts-based FSH Society and the FSHD Champions ask everyone to join World FSHD Day to raise awareness for facioscapulohumeral muscular dystrophy (FSHD). FSHD patients, their families and their supporters as well as advocacy organizations will come together to urge a cure for one of…

A former investigational drug initially designed to target cancer was found to have therapeutic potential for Duchenne muscular dystrophy (DMD), according to an article published in the scientific journal Molecular Therapy. The study, “SU9516 Increases α7β1 Integrin and Ameliorates Disease Progression in the mdx Mouse Model of Duchenne…

Advocates from Save Our Sons Duchenne Foundation (SOS), in partnership with Parent Project Muscular Dystrophy (PPMD), have launched Australia’s first patient-led registry to increase clinical trial recruitment in Duchenne muscular dystrophy (DMD). The initiative was inspired by PPMD’s DuchenneConnect patient registry, which since 2007 has provided data…

Sarepta Therapeutics is recruiting up to 99 boys with Duchenne muscular dystrophy (DMD) to test two new compounds, SRP-4045 and SRP-4053, in a Phase 3 clinical trial. The ESSENCE study is a double-blind, placebo-controlled, multi-center Phase 3 clinical trial (NCT02500381) with an open-label extension period, evaluating the two drugs’…

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…

A new clinical-stage biotech, Myonexus Therapeutics, is developing gene therapies for limb-girdle muscular dystrophies (LGMDs). Launch plans include the accelerated development of five investigational gene therapies that have the potential to become the first standard-of-care therapies for LGMDs. Myonexus’ pipeline includes three clinical-stage gene therapy programs (LGMD2E, LGMD2D, and…

The U.S. Food and Drug Administration (FDA) scheduled a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting with PTC Therapeutics to review the company’s new drug application (NDA) for Translarna (ataluren) to treat patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). The meeting will take place on Sept. 28, the company…

Genetic screening for the presence of mutations that characterize Duchenne and Becker muscular dystrophies (DMD and BMD) in women before conception can help determine the risk their offspring will develop either disease, according to a Polish study published in the Journal of Obstetrics Gynaecology Research. The study, “Prenatal diagnosis of Duchenne…