News

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…

More than 420 “Fill the Boot” events will raise money throughout Labor Day weekend to advance research and care for muscular dystrophy. The campaign results from a partnership between the Muscular Dystrophy Association (MDA) and the International Association of Fire Fighters (IAFF). The firefighters go to…

Deleting exon 51 in the DMD gene — which mimics exon 51 skipping therapies — reversed all signs of Duchenne muscular dystrophy (DMD) in a pig model lacking exon 52, a severe disease-causing mutation in people, a study reported. Pigs missing both exon 51 and 52 had normalized dystrophin…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AOC 1044, an experimental exon 44-skipping therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Avidity Biosciences. Orphan drug designation is given to therapies with the potential to treat rare disorders, those that affect fewer…