Fulcrum Therapeutics' promising treatment for Sickle Cell Disease and their strong financial position, despite setbacks.
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.
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Communication between body muscle and pituitary gland may play unexpected role in female fertilityA newly discovered line of communication between body muscle and the pituitary gland may play an unexpected role in female ...
A newly discovered line of communication between body muscle and the pituitary gland may play an unexpected role in female fertility, according to a new study. Researchers found that a protein ...
The Muscular Dystrophy Association (MDA) is honoring a longtime researcher and an LGMD patient advocate with its 2025 Legacy ...
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