The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CRD-003, an investigational gene therapy for…
Articles by Marisa Wexler, MS
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…
A Phase 1/2 clinical trial testing SAR446268, an experimental gene therapy for myotonic dystrophy type 1 (DM1), is enrolling…
Note: This story was updated Sept. 10, 2025, to correct the secondary headline from Columbia University to the University of…
Researchers have developed a way to enhance the efficacy of exon-skipping therapies for Duchenne muscular dystrophy (DMD), and the…
Preliminary data are expected this year for a Phase 1/2 clinical trial testing the investigational therapy SRP-1003 in people with…
The steroid therapy Agamree (vamorolone), which is used for helping to preserve muscle function in people with Duchenne…
A protein called MYOD, which is known to control the growth of muscle stem cells during muscle repair, has long…
Enrollment is now ongoing in a first-in-human trial testing EPI-321, an epigenetic therapy designed to address the root cause of…
Note: This story was updated July 30, 2025, to include data from part two of the EMBARK trial. Sarepta…