Sarepta Therapeutics' Elevidys for DMD shows revenue potential, with market cap justified at $11.4bn and potential for >50% ...

DYNE-101 eased myotonia and led to other functional improvements for people with myotonic dystrophy type 1 (DM1) in a ...

Currently, the DMD treatment landscape includes Sarepta’s Elevidys (delandistrogene moxeparvovec), the first FDA-approved ...

GIVI-MPC, a stem cell therapy for certain types of muscular dystrophy, has been granted orphan drug status for treating ...

Ips Heart Inc. has been awarded orphan drug designation by the FDA for its GIVI-MPC stem cell therapy for Becker muscular dystrophy. GIVI-MPC has the unique ability to create new muscle with full ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported preliminary* fourth quarter and f ...

Entering 2025 having demonstrated rapid translation of genetic insights to positive clinical data; advancing clinically validated and multi-modal pipeline with the potential to treat well over 100 mil ...

Entering 2025 having demonstrated rapid translation of genetic insights to positive clinical data; advancing clinically validated and multi-modal ...

IPS HEART has been awarded Orphan Drug Designation (ODD) by the FDA for GIVI-MPCs based on its unique ability to create new ...