News

A Phase 2 clinical trial testing the investigational oral medication EDG-5506 in people with Becker muscular dystrophy (BMD) is expanding, with plans to enroll 120 additional adults with the genetic disorder. According to EDG-5506’s developer Edgewise Therapeutics, enrollment in the expanded study is ongoing. The company is hosting…

AMO-02 (tideglusib), an experimental oral therapy for certain muscular dystrophies, showed promise in boosting motor and cardiac muscle health in a mouse model of Duchenne muscular dystrophy (DMD). According to AMO Pharma, the treatment’s developer, AMO-02 also improved the animals’ metabolism and cognitive abilities. “These studies…

Covering topics such as mobility equipment and the role of physical therapy, a new, free, online CureDuchenne course seeks to provide guidance and support to Duchenne muscular dystrophy (DMD) caregivers. Specifically, the virtual CureDuchenne Caregiver Course seeks to help caregivers of those newly diagnosed with DMD —…

The conditional approval of Translarna (ataluren) in Europe as a treatment for Duchenne muscular dystrophy (DMD) — in place for nearly a decade — should now not be renewed, a committee of the European Medicines Agency (EMA) has recommended. The EMA’s Committee for Medicinal Products for Human Use…

Fulcrum Therapeutics has completed patient enrollment for its Phase 3 clinical trial evaluating losmapimod as a potential treatment for people with Facioscapulohumeral muscular dystrophy (FSHD). The study, dubbed REACH (NCT05397470), is evaluating the efficacy and safety of losmapimod against a placebo in FSHD adult patients across…

Entrada Therapeutics has awarded $25,000 grants to three U.S. nonprofits to aid in their efforts to combat healthcare disparities in the Duchenne muscular dystrophy (DMD) community. The goal of these nonprofits, as inaugural recipients of the biopharmaceutical company’s DREAMS — Diversity, Representation, Equity and Advocacy Matters — grant…

The European Medicines Agency (EMA) has started reviewing an application to market givinostat for Duchenne muscular dystrophy (DMD). “Now that our market authorization application is under review, we are looking forward to working with the regulatory authorities to bring this treatment option to patients,” Paolo Bettica, MD, PhD,…

Pamrevlumab, an antibody in FibroGen’s pipeline, did not improve motor skills in boys with Duchenne muscular dystrophy (DMD), ages 6 to 11, who could walk, according to top-line data from a Phase 3 study. The LELANTOS-2 (NCT04632940) trial tested against a placebo pamrevlumab’s safety and how…

September will be a busy month for patients, supporters, doctors, and researchers in the muscular dystrophy (MD) community as they recognize National Muscular Dystrophy Awareness Month. Raising awareness and critical funds for the more than 300,000 families across the country with MD and related neuromuscular diseases is the goal…

Blocking the activation of a channel in mitochondria, the cells’ powerhouses, may be a promising way to stop muscular dystrophy (MD) progression in a mouse model of the disease. “We have isolated the primary disease-causing component of muscular dystrophy to the mitochondrial permeability pore,” Jeffery Molkentin, PhD, co-executive director…