PTC Therapeutics recently revealed the 5 winners of its inaugural global awards program, the STRIVE Awards (Strategies to Realize Innovation, Vision and Empowerment). These awards seek to help not-for-profit organizations that cater to the Duchenne muscular dystrophy (DMD) community. PTC made the announcement during the international World Duchenne Awareness Day held on Monday,…
The FSH Society, a nonprofit, patient-driven organization supporting research and education for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy, recently announced it will be the beneficiary of a number of fundraising events during this fall. The events which are aimed to raise awareness for the FSH…
BioMarin Pharmaceutical, Inc., a company that develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions, has officially launched kNOWyourDuchenne, a program dedicated to assisting families and clinicians gain access to genetic testing for patients with Duchenne muscular dystrophy (DMD). This first-of-its-kind program will work to pinpoint specific genetic mutations linked to…
Duchenne muscular dystrophy (DMD) is the most frequently inherited human myopathy and, as the name implies, is characterized by wasting of the muscles throughout the body. The disease is caused by a mutation of the dystrophin gene at the X chromosome and is characterized by muscle weakness of the voluntary muscles. The hips, pelvic area, thighs,…
Arizona-based Critical Path Institute (C-Path), a leading non-profit organization committed to accelerate and reduce developmental costs of medical products, recently announced it has formed its 10th consortium: The Duchenne Regulatory Sciences Consortium (D-RSC). Together with Parent Project Muscular Dystrophy (PPMD), the new consortium will utilize C-Path’s tested consensus science model to find a cure for…
Eleven year-old mountaineer, Tyler Armstrong, first fell in love with conquering the outdoors when he was 7 years old. Today, he is determined to climb for a cause, on behalf of those who cannot. Tyler plans to be the youngest climber to take on Mt. Everest to help raise funds for…
In a recent study published in the Journal of Pediatric Ophthalmology and Strabism, a team of researchers from Turkey found that ophthalmological problems are commonly seen in children with muscular dystrophies. Muscular dystrophy includes a group of diseases that cause progressive weakness and loss of muscle mass,…
Santhera Pharmaceuticals, a Swiss pharmaceutical company specialized in developing and commercializing breakthrough treatments for orphan mitochondrial and neuromuscular diseases, announced that it has dosed the first patient in its CALLISTO Phase I trial that aims to evaluate the safety and tolerability profile of orally-stable omigapil for Congenital Muscular Dystrophy (CMD). All necessary participants have…
Researchers have been exploring the use of stem cell therapeutics for the treatment of severe conditions such as type 1 diabetes, Alzheimer’s disease and Muscular Dystrophy. There are several types of stem cells that scientists think may be used in different ways to develop treatments for muscular dystrophy. The main stem-cell-based approaches…
A scientist who helped develop a pioneering treatment recently expressed her frustration after the NHS refused funding of Translarna for the treatment of Duchenne Muscular Dystrophy. Translarna has recently become the first Duchenne Muscular Dystrophy targeting therapy to receive approval by the European Medicines Agency (EMA). The drug is available in many…
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