News

Benitec Biopharma is taking steps to get the needed regulatory clearances to start clinical testing in Europe and the U.S. of BB-301, its experimental gene therapy for oculopharyngeal muscular dystrophy or OPMD, a type of the genetic disease that typically first affects the eyelids and the throat. The…

A team of researchers received a $3-million grant from the Chan Zuckerberg Initiative to create a comprehensive blueprint of healthy skeletal muscle cells — whose dysfunction is associated with several rare muscle diseases, such as muscle dystrophy — as children grow. Muscular dystrophy comprises a group of more…

A myriad of activities are afoot to mark National Muscular Dystrophy Awareness Month, observed each September. The events aim to raise funds for, and heighten awareness of, muscular dystrophy (MD) and related neuromuscular disorders thought to affect more than 300,000 families across the U.S. Awareness days are set for…

The European Commission (EC) has granted orphan designation to AOC 1001, Avidity Biosciences’ investigational therapy for myotonic dystrophy type 1 (DM1). This designation is given to investigational therapies that have the potential to treat rare, life-threatening, or very serious diseases that affect less than five in 10,000 patients…

Cumberland Pharmaceuticals is recruiting boys and men for a Phase 2 study evaluating the safety, efficacy, and duration of effect of its oral ifetroban therapy for cardiomyopathy — a disease of the heart muscle — linked to Duchenne muscular dystrophy (DMD). The study, called FIGHT DMD (NCT03340675),…

MRI scans of muscle can be used as a biomarker to assess disease progression in Duchenne muscular dystrophy (DMD) and to support the evaluation of different therapies in clinical trials, an analysis showed. “The results of this study provide further support for the use of imaging as an objective,…

The U.S. Social Security Administration (SSA) has added Duchenne muscular dystrophy (DMD) to its Compassionate Allowances (CAL) program list, accelerating the disabilities application process for adults with this genetic disorder. The program is designed to identify severe medical diseases and conditions that automatically meet the SSA’s standards for…

The U.S. Food and Drug Administration (FDA) has granted fast track status to EDG-5506, Edgewise Therapeutics’ investigational oral therapy for Becker muscular dystrophy (BMD). This designation is given to experimental treatments that are intended to treat serious conditions and fill an unmet medical need. It confers certain benefits, like…

PepGen has raised funding totaling $112.5 million to advance toward the clinic its enhanced delivery oligonucleotide (EDO) lead programs in Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The funding round keeps PepGen on track to start Phase 1 clinical trials in 2022 for DMD, and…

Losmapimod, a potential oral treatment for facioscapulohumeral muscular dystrophy (FSHD), missed its main goal in a recent Phase 2 clinical trial, but its developer, Fulcrum Therapeutics, sees this as a constructive failure. Lessons learned will help to shape losmapimod’s continued development, which is supported by the secondary…