News

Sarepta Therapeutics, Inc., a biopharmaceutical company focused on the development of innovative RNA-based therapeutics, recently announced that the U.S. Food and Drug Administration (FDA) has filed the New Drug Application (NDA) for the company’s eteplirsen. A NDA is the vehicle through which drug sponsors formally propose FDA to approve a new…

The Muscular Dystrophy Association (MDA) on August 26th announced its awarding of $10 million in new grants to scientists conducting leading-edge research on muscular dystrophy, ALS and related muscle-debilitating diseases. Through its investigator-initiated application process, MDA had received 350 grant requests — a record number in its 65-year history of…

A study recently published in the journal Scientific Reports reported the development of a novel in vitro model of the early pathogenesis of Duchenne muscular dystrophy (DMD) using stem cells. The study is entitled “Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human…

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,…

BioMarin Pharmaceutical Inc. has recently announced that the United States Food and Drug Administration (FDA) attributed a rare pediatric disease designation for drisapersen, a potential treatment for individuals suffering with Duchenne Muscular Dystrophy (DMD) who are responsive to exon 51 skipping treatment. Drisapersen has been previously granted Orphan Drug and Fast…

In a new study entitled “Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells” a team of scientists developed an induced pluripotent stem cell model to study Duchenne muscular dystrophy and discover new therapeutic drugs. The study was published in the journal…

In a new study entitled “Differential Isoform Expression and Selective Muscle Involvement in Muscular Dystrophies”researchers performed a microarray analysis on human muscles to identify the molecular cause underlying the selectivity of muscles affected in muscular dystrophies. Their findings suggest that overexpression of mutated genes in specific muscles are…

A new 77-page GBI Research report entitled “Frontier Pharma: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy – Identifying and Commercializing First-in-Class Innovation” identifies and highlights a highly innovative and diverse pipeline of products for treating Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) Currently in the pipeline are 84…

In a new study entitled “Genome-wide Mechanosensitive MicroRNA (MechanomiR) Screen Uncovers Dysregulation of their Regulatory Networks in the mdm Mouse Model of Muscular Dystrophy” researchers performed a genome-wide study to identify whether mechanosensitive microRNAs are deregulated and contribute to muscular dystrophies pathogenesis. The study was published in…

Pfizer is currently recruiting participants to enroll a phase 2 clinical trial that intends to assess the safety, efficacy and pharmacological parameters of its product, PF-06252616, in boys with Duchenne muscular dystrophy (DMD) who are able to walk (ambulatory). The clinical trial is registered under the identifier NCT02310763…