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.

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

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.

Muscular dystrophy can appear in infancy up to middle age or later, and its form and severity are determined in part by the age at which it occurs. Some types of muscular dystrophy typically ...

Dystrophy is any condition in which a part of the body weakens or wastes away. In muscular dystrophy, the weakness is in the muscles. An inherited genetic mistake prevents the body from making a ...

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the global licensing and collaboration agreement with Sarepta Therapeutics (NASDAQ: SRPT) announced on November 26, 2024, has now ...

The Muscular Dystrophy Association (MDA) is honoring a longtime researcher and an LGMD patient advocate with its 2025 Legacy ...