Fulcrum Therapeutics' promising treatment for Sickle Cell Disease and their strong financial position, despite setbacks.

Vergara was diagnosed with facioscapulohumeral muscular dystrophy in 2002. Five years later, he and his mother moved from their hometown, Cartagena, Colombia, to the United States, where he had ...

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule.

candidate for myotonic dystrophy type 1 (DM1), and del-brax for facioscapulohumeral muscular dystrophy (FSHD). All three candidates are based on the same TfR1-targeting antibody component.

From left, Craig Meyer, Silvia Blemker and Joseph Hart are the University of Virginia’s Innovators of the Year. The UVA ...

Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...