- DYNE-101 in DM1: Dyne plans to initiate global Registrational Expansion Cohort of ACHIEVE trial with registrational dose of 6.8 mg/kg Q8W following study data showing splicing correction and robust ...

– U.S. FDA has confirmed that screening and dosing may proceed in Study SRP-9005-101 for LGMD2C/R5 – Enrollment and dosing completed in Study SRP-9004-102 for LGMD2D/R3 – Data expected for SRP-9003 ...

Multiple biological pathways go awry to cause disease. Cambridge startup Catabasis is developing a platform to simultaneously modulate multiple targets in a disease pathway, with a focus on diseases ...

ROCKFORD, Ill. (AP) -- Authorities in Rockford have identified the city's sixth homicide victim of the year as Katherine Means, a 50-year-old woman who suffered from muscular dystrophy. Means was ...