A treatment combination of two blood pressure medicines can help slow heart function decline in males with Duchenne muscular…
Articles by Patricia Inácio, PhD
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…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
The Muscular Dystrophy Association (MDA) has named Keisha Greaves, a fashion entrepreneur and neuromuscular disease advocate from Cambridge, Massachusetts,…
The first patient has been dosed in a Phase 2 trial testing BBP-418, a potential first oral disease-modifying therapy…
Santhera Pharmaceuticals has obtained the worldwide rights to vamorolone (VBP15), an experimental therapy for Duchenne muscular dystrophy (DMD),…
This year, the Muscular Dystrophy Association (MDA) will celebrate MDA National Muscular Dystrophy Awareness Month, held every September, with…
The U.S. Food and Drug Administration (FDA) is maintaining the clinical hold on the Phase 1/2 trial testing Solid Biosciences’…