Lindsey Shapiro, PhD, science writer —

Ahead of pending regulatory decisions for vamorolone — a treatment for Duchenne muscular dystrophy (DMD) now under review in the U.S. and Europe — Catalyst Pharmaceuticals has fully acquired the commercial rights to the therapy in North America. The finalized deal comes about a month after Catalyst…

The Muscular Dystrophy Association (MDA) has launched the Gene Therapy Support Network (GTx) that will offer resources and guidance about approved gene therapies for people living with neuromuscular diseases. Its launch comes shortly ahead of a possible U.S. approval of SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics‘ experimental…

Nearly half of the 24 children with myotonic dystrophy type 1 (DM1) in a French study showed signs of poorer nighttime respiration, reflected by rising carbon dioxide (CO2) levels. These increases in CO2 did not significantly associate with the presence of sleep apnea — when breathing repeatedly starts and…

Pelvic floor disorders, including urinary and anal incontinence, are common in women with myotonic dystrophy type 1 (DM1) and a cause of significant distress, according to a recent study in Canada. “This study highlights the importance of screening these symptoms in a clinical setting and will help develop interventions…

The U.S. Food and Drug Administration (FDA) has placed a hold on clinical testing of ENTR-601-44, Entrada Therapeutics‘ investigational therapy for people with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 44 skipping. The decision was in response to the company’s investigational new drug (IND) application,…

Analyzing the type of mutation in the DMD gene combined with assessing motor milestones may help better distinguish Duchenne muscular dystrophy (DMD) from Becker muscular dystrophy (BMD) early in life, a study found. Combining both approaches had better predictive power than using either alone, and could mean that children will…

The first patient has been dosed in a pilot study evaluating vamorolone as a potential treatment for Becker muscular dystrophy (BMD). The steroid medication has been under development by ReveraGen BioPharma as a possible therapy for other types of muscular dystrophy. Now, a Phase 2 trial…

The U.S. Food and Drug Administration (FDA) has given the go-ahead for the first-in-human dosing of Cure Rare Disease’s (CRD) investigational genome-editing therapeutic for Duchenne muscular dystrophy (DMD). Dosing of a single patient with the treatment, dubbed CRD-TMH-001, will happen soon at the University of Massachusetts Chan Medical…

Treatment with dystrophin expressing chimeric (DEC) cells was safe and led to long-term cellular and functional improvements in the heart, respiratory, and leg muscles of a mouse model of Duchenne muscular dystrophy, a study found. The findings support “future clinical application of DEC as a safer and potentially more…

The first healthy volunteer has been dosed with an investigational exon-skipping therapy, called PGN-EDO51, for Duchenne muscular dystrophy (DMD) in a Phase 1 trial, the treatment’s developer, PepGen, has announced. The single-ascending dose trial is investigating the treatment’s safety and tolerability in about 40 healthy adult males in Canada.