Abstract Title: Safety and Efficacy of DYNE-101 in Adults with DM1: Phase 1/2 ACHIEVE Trial Data Date and Time: Wednesday, March 19, at 12:30-12:45 p.m. CT Presenter: James Lilleker M.D., Neurologist, ...

Facioscapulohumeral muscular dystrophy (FSHD) is a genetic condition that affects muscle function, often making everyday tasks — like smiling or lifting one’s arms — difficult. For ...

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.