Note: This story was updated Nov. 5, 2025, to clarify that enrollment in the follow-up study is ongoing. Dosing has…
Articles by Marisa Wexler, MS
In a deal worth roughly $12 billion, pharmaceutical giant Novartis has agreed to acquire Avidity Biosciences, to develop…
Use of the oral therapy BBP-418 appeared to improve walking ability and lung function in people with limb-girdle muscular…
Three boys with Duchenne muscular dystrophy (DMD) who were given the experimental gene therapy GNT0004 in a clinical trial…
Health Canada has approved Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older,…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CRD-003, an investigational gene therapy for…
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…