Treatment with BBP-418 improved walking ability among people with limb-girdle muscular dystrophy type 2i (LGMD2i) in a Phase 2…
Articles by Marisa Wexler, MS
A potentially pivotal Phase 3 clinical trial called HOPE-3, which will test the effectiveness of the experimental cell therapy…
A protein called GCN5 is vital for normal muscle health and helps to coordinate the expression of several important muscle…
Children with Duchenne muscular dystrophy (DMD) who had been given a placebo in a clinical trial experienced a marked…
Elamipretide, an investigational therapy designed to increase the activity of mitochondria, may boost the effectiveness of exon-skipping therapies for…
Atamyo Therapeutics has been given the go-ahead to start clinical testing of ATA-100, the company’s investigational gene therapy for a specific…
Santhera Pharmaceuticals and ReveraGen BioPharma are gearing up to submit a new drug application (NDA) asking for U.S.
Atamyo Therapeutics is seeking approvals in Europe to launch its first trial testing ATA-100, its investigational gene therapy for a…
The COVID-19 pandemic has had a substantial impact on the mental health and quality of life of people affected by…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to AOC 1001, an investigational treatment for…