Fulcrum Therapeutics plans to launch a Phase 3 trial of losmapimod, a potential oral treatment for facioscapulohumeral muscular…
Articles by Patricia Inácio, PhD
Muscular Dystrophy News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to muscular…
A branch of the U.S. Food and Drug Administration (FDA) known as the Division of Cardiology and Nephrology has given…
A Phase 1 trial of SRP-9001, Sarepta Therapeutics‘ investigational gene therapy, is recruiting 3-year-old boys with Duchenne muscular dystrophy…
Tamoxifen, commonly used to treat breast cancer, failed to meet its primary goal — delaying disease progression — in a Phase…
CureDuchenne has teamed up with Brigham and Women’s Hospital, in Boston, to launch the nation’s first free newborn…
A treatment combination of two blood pressure medicines can help slow heart function decline in males with Duchenne muscular…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
For the first time, a new mouse model has been shown to mimic the heart disease features — specifically, to…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…