Marisa Wexler, MS,  senior science writer—

Exon-skipping therapies Amondys 45 (casimersen) and Vyondys 53 (golodirsen) show “positive and encouraging trends” for people with Duchenne muscular dystrophy (DMD), even though top-line results from a Phase 3 trial testing the treatments showed the study failed to meet its main goal, developer Sarepta Therapeutics said. Results from…

Note: This story was updated Nov. 5, 2025, to clarify that enrollment in the follow-up study is ongoing. Dosing has begun in a long-term follow-up study testing Satellos Bioscience’s experimental oral therapy SAT-3247 in men with Duchenne muscular dystrophy (DMD). The study — called LT-001 (NCT06867107) —…

In a deal worth roughly $12 billion, pharmaceutical giant Novartis has agreed to acquire Avidity Biosciences, to develop several promising experimental therapies for various types of muscular dystrophy. The core of the acquisition is Avidity’s antibody-oligonucleotide conjugate (AOC) platform. This innovative technology attaches a small piece of…

Use of the oral therapy BBP-418 appeared to improve walking ability and lung function in people with limb-girdle muscular dystrophy type 2i (LGMD2i) — characterized by muscle wasting at the shoulders and the hips — in a Phase 3 clinical trial, according to new interim data announced by developer…

Three boys with Duchenne muscular dystrophy (DMD) who were given the experimental gene therapy GNT0004 in a clinical trial were seeing sustained improvements in motor function and reductions in markers of muscle damage two years after the one-time treatment. Genethon, GNT0004’s developer, presented the findings at the European…

Health Canada has approved Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older, making Agamree the first approved DMD treatment in Canada. Canada’s approval is the sixth for Agamree to treat DMD. The therapy is approved in the U.S., the U.K.,…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CRD-003, an investigational gene therapy for limb-girdle muscular dystrophy type 2i/R9 (LGMDR9). The FDA gives this designation to experimental therapies designed to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S.

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to NS-051/NCNP-04, an exon-skipping therapy designed to treat certain people with Duchenne muscular dystrophy (DMD). The FDA gives this designation to experimental treatments designed to improve care for rare disorders, specifically conditions affecting fewer than 200,000 people…

A Phase 1/2 clinical trial testing SAR446268, an experimental gene therapy for myotonic dystrophy type 1 (DM1), is enrolling patients. The trial, dubbed BrAAVe (NCT06844214), is expected to enroll approximately 32 DM1 patients aged 10-50. The study is actively recruiting participants at sites in Florida, New York,…

Note: This story was updated Sept. 10, 2025, to correct the secondary headline from Columbia University to the University of Iowa. Columbia University awarded the 2025 Louisa Gross Horwitz Prize to three scientists whose research has helped lay the groundwork to develop new treatments for Duchenne muscular dystrophy (DMD). This…