Margarida Maia, PhD, science writer —

IPS Heart has received rare pediatric drug designation for its GIVI-MPC and ISX9-CPC, two experimental single-course stem cell therapies for Duchenne muscular dystrophy (DMD). The designation is given by the U.S. Food and Drug Administration (FDA) to spur treatments for any rare disease that can be serious or…

A form of congenital muscular dystrophy caused by mutations in a gene called GGPS1 may not always lead to hearing loss, contrary to what has been described previously, according to a recent study. The report, “GGPS1-associated muscular dystrophy with and without hearing loss,” was published as a brief…

Transferring a healthy version of the Large1 gene into adult mice with severe congenital muscular dystrophy restored the function of muscles involved in movement and breathing, a study found. The use of this gene therapy translated into an extended lifespan for the mice, which had congenital muscular dystrophy —…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ATA-100, a one-time gene therapy being developed by Atamyo Therapeutics for a specific form of limb-girdle muscular dystrophy (LGMD) called 2I or R9 (LGMD2I/R9). In Europe, the therapy also was named an orphan drug by…

Heart problems can progress quickly in people with myotonic dystrophy type 2 (MD2) or facioscapulohumeral muscular dystrophy type 1 (FSHD1), even in those showing no symptoms of cardiac disease, a study reported. “MD2 and FSHD1 patients should be carefully followed-up to identify early development of remodeling and potential risks for the development…

Treatment with SGT-001 — Solid Biosciences’ gene therapy candidate for Duchenne muscular dystrophy (DMD) — improves lung function, according to data from the first six patients enrolled in the ongoing IGNITE DMD clinical trial. The improvements, seen one year after a single infusion of the SGT-001 gene therapy into…

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),…

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…