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…
The name mitochondrial myopathy comes from the specific part of the cell the disease affects, called the mitochondria, which are small energy factories found inside most cells in our body. MORE: Remembering Mattie Stepanek “Myopathy” comes from two words: “myo” meaning muscle, and “pathy” meaning disease. If you put those…
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…
Mattie Stepanek’s story is well-known in the MD community. Mattie was the youngest of four children, all of whom died in childhood from complications due to the genetic neuromuscular disorder, dysautonomic mitochondrial myopathy. MORE: Musclar dystrophy quote: Mattie Stepanek Mattie touched the hearts of millions of Americans and was a…
Using cannabis or marijuana for medicinal purposes is a hot topic right now among patients and health practitioners. It’s regularly touted as a pain reliever for many chronic illnesses. According to the National Cancer Institute, here are some of the facts about medical marijuana and its suggested uses: It’s…
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…
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