The U.S. Food and Drug Administration (FDA) has cleared Avidity Biosciences to start a Phase 1/2 clinical trial testing…
Articles by Marisa Wexler, MS
The U.S. Food and Drug Administration (FDA) has granted fast track designation to LION-101, an investigational gene therapy that…
Vita Therapeutics announced that it has raised $32 million in financing to advance its work into cell-based therapies for…
With current corticosteroid use, people with Duchenne muscular dystrophy (DMD) typically lose their walking ability by their early teens,…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for…
Asklepios BioPharmaceutical (AskBio) is planning to launch a Phase 1/2 clinical study to evaluate LION-101, its investigational gene therapy…
Sarepta Therapeutics‘ investigational gene therapy SRP-9001 showed early signs of biologic efficacy in muscle biopsies from 11 boys…
Two more participants have been dosed in the IGNITE DMD clinical trial, bringing to eight the total number of Duchenne…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to losmapimod, a potential treatment for…
Jesse’s Journey, a Canadian nonprofit founded by parents of a child with Duchenne muscular dystrophy (DMD), has given…