Elamipretide, an investigational therapy designed to increase the activity of mitochondria, may boost the effectiveness of exon-skipping therapies for…
Articles by Marisa Wexler, MS
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…
Note: This story was updated Oct. 8, 2021, to clarify that Sarepta did not confirm previously announced plans to…
Treatment with the investigational cell therapy CAP-1002 significantly improved arm and heart function in boys and young men at advanced…
BBP-418, an investigational disease-modifying medication that aims to improve muscle strength and function in people with limb-girdle muscular…
A new viral vector that is better than conventional vectors — tools designed to deliver genetic material into cells —…