News

A recent article published in the Annals of Neurology describes promising results from a Phase 2b study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). The study, titled “Longitudinal effect of eteplirsen vs. historical control on ambulation in DMD,” is the result of a collaborative effort between researchers from…

Muscular dystrophies (MDs) cause progressive weakness of muscles in young boys. Although treatments alleviate the symptoms and slow down disease progression, there is no cure for MDs. Research in regenerative medicine may offer novel therapies to restore muscle function in MDs patients as shown by scientists at KU Leuven University in…

Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene expressed in differentiated myofibers. Researchers at The Ottawa Hospital and the University of Ottawa recently discovered that muscle stem cells also express dystrophin, affecting the generation of functional muscle fibers. Their discovery could revolutionize understanding of the disease and eventually…

Ringo, a golden retriever, has made an important contribution to science by helping researchers discover a gene that inhibits the consequences of dystrophin loss in Duchenne muscular dystrophy (DMD). Even though Ringo carried the classic DMD mutation, he and one of his male offsprings had a variant in the Jagged1 gene that allowed…

aTyr Pharma, Inc., has announced an expansion of its Phase 1b/2 clinical trial to continue the development and evaluation of efficacy and safety of the Resolaris clinical program, for the treatment of early onset facioscapulohumeral muscular dystrophy (FSHD) patients. Facioscapulohumeral muscular dystrophy (FSHD) is a genetic muscular disorder, characterized by progressive muscle…

ISIS Pharmaceuticals, a company focusing on the development of RNA-targeted drug discovery using its antisense technology platform, announced it earned a $2.8 million milestone payment from Biogen to continue developing the ongoing Phase 1/2a study of ISIS-DMPK-2.5Rx for the treatment of myotonic dystrophy type I (DM1) patients. This payment stems from a collaboration with Biogen and…

Ironwood Pharmaceuticals, Inc., recently announced the beginning of a Phase Ib clinical trial of IW-1973 and a Phase Ia clinical trial of IW-1701, both soluble guanylate cyclase (sGC) stimulators from a wide-ranging, pharmacologically distinct portfolio of sGC composites discovered by the company. The clinical results intend to determine dose selections along with the main…

San Rafael, California-based biotechnology company BioMarin Pharmaceutical’s experimental drug drisapersen, which is under development as a new treatment for Duchenne muscular dystrophy (DMD), is ready to enter the market pending approval by the U.S. Food and Drug Administration (FDA). According to The Muscular Dystrophy Association (MDA) — a global…

Recent research has demonstrated that early bisphosphonate treatment of a mice model of Duchenne muscular dystrophy (DMD) had some positive effects on bone integrity and skeletal muscle strength. The research paper, entitled “Positive effects of bisphosphonates on bone and muscle in a mouse model of Duchenne muscular dystrophy,” was…

Professor Liza Pon, PhD, a leading researcher at the department of pathology and cell biology at Columbia University in New York, was recently awarded a $300,000, three-year research grant from the Muscular Dystrophy Association (MDA) to continue her work in congenital muscular dystrophy (CMD). In a recent interview conducted by…