A first patient has been dosed in Denmark as part of a Phase 1/2 trial of ATA-100, Atamyo…
Articles by Patricia Inácio, PhD
Treatment with Exondys 51 (eteplirsen) outperformed standard of care therapy at delaying respiratory decline in boys with Duchenne muscular…
A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases…
The first patient has been dosed in a pivotal U.S. Phase 3 study evaluating the investigational cell therapy CAP-1002…
A Phase 2 clinical trial testing Edgewise Therapeutics’ investigational oral therapy EDG-5506 is recruiting boys and men with…
Using the results of a New York State pilot testing program as evidence, Parent Project Muscular Dystrophy (PPMD) submitted…
FibroGen has completed patient enrollment for its Phase 3 trial evaluating pamrevlumab in combination with corticosteroids as a…
New data from a Phase 1/2 clinical trial of an exon-skipping gene therapy suggest it may help preserve muscle function…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to elamipretide for treating Duchenne muscular…
Santhera Pharmaceuticals and ReveraGen BioPharma will seek approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD) in the U.S. this…