Marisa Wexler, MS,  senior science writer—

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…

Researchers have developed a way to enhance the efficacy of exon-skipping therapies for Duchenne muscular dystrophy (DMD), and the approach showed promise in a mouse model of the disease. The study, “A Combinatorial Oligonucleotide Therapy to Improve Dystrophin Restoration and Dystrophin-Deficient Muscle Health,” was published in Molecular Therapy…

Preliminary data are expected this year for a Phase 1/2 clinical trial testing the investigational therapy SRP-1003 in people with myotonic dystrophy type 1 (DM1), according to an announcement from Sarepta Therapeutics. The trial recently hit one of two prespecified enrollment targets, prompting a review of safety data.

The steroid therapy Agamree (vamorolone), which is used for helping to preserve muscle function in people with Duchenne muscular dystrophy (DMD), will be sold and promoted in Turkey by Gen İlaç ve Sağlık Ürünleri San. ve Tic. A.Ş. (GEN), per an agreement with Santhera Pharmaceuticals. “As leaders…

A protein called MYOD, which is known to control the growth of muscle stem cells during muscle repair, has long been known to be able to turn on genes, but a recent study has now found that MYOD can also turn off certain genes. According to researchers, the findings may…

Enrollment is now ongoing in a first-in-human trial testing EPI-321, an epigenetic therapy designed to address the root cause of facioscapulohumeral muscular dystrophy (FSHD), a disease type in which symptoms like muscle weakness typically become apparent by adulthood. The Phase 1/2 clinical trial (NCT06907875) is expected to enroll…

Note: This story was updated July 30, 2025, to include data from part two of the EMBARK trial. Sarepta Therapeutics will soon resume shipping Elevidys (delandistrogene moxeparvovec-rokl) for ambulatory Duchenne muscular dystrophy (DMD) patients, following a review of safety data by the U.S. Food and Drug Administration (FDA),…