The U.S. Food and Drug Administration (FDA) has placed a hold on Dyne Therapeutics’ application to launch a clinical…
Articles by Steve Bryson, PhD
A young man with Duchenne muscular dystrophy (Â鶹Éç), who was participating in Pfizer’s Phase 1B open-label clinical trial…
RGX-202, a one-time experimental gene therapy for the treatment of Duchenne muscular dystrophy (Â鶹Éç), has been granted orphan…
Translarna (ataluren) treatment delayed the loss of walking abilities by more than five years in boys with Duchenne…
MRI scans of muscle can be used as a biomarker to assess disease progression in Duchenne muscular dystrophy (Â鶹Éç)…
Oral losmapimod, an investigational treatment for people with facioscapulohumeral muscular dystrophy (FSHD), showed clinically relevant benefits over a…
An analysis of microRNA molecules isolated from the blood of people with Duchenne muscular dystrophy (Â鶹Éç) found alterations related…
Top-line data from the VISION-Â鶹Éç Phase 2b clinical trial evaluating vamorolone…
Pfizer has announced a delay in opening U.S. sites for its Phase 3 clinical trial CIFFREO, evaluating…
Sarepta Therapeutics has announced positive results from a global study evaluating its investigational treatment…