Margarida Maia, PhD,  science writer—

In the U.S., only a limited number of patients will enter Sarepta Therapeutics‘ ENVISION, a Phase 3 study that’s part of the company’s strategy to gain global approval of SRP-9001 (delandistrogene moxeparvovec) as a gene therapy for Duchenne muscular dystrophy (DMD). These DMD patients will be non-ambulatory, or…

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on an application to launch a Phase 1 study of PGN-EDODM1, an experimental medicine by PepGen for people with myotonic dystrophy type 1 (DM1). A clinical hold is an order issued by the FDA to delay…

The U.S. Food and Drug Administration (FDA) has pushed back its decision on SRP-9001 (delandistrogene moxeparvovec), an experimental gene therapy for Duchenne muscular dystrophy (DMD). A decision is now expected by June 22, according to a press release. If the agency decides to approve SRP-9001, it’s expected to be…

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…