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

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported ...

Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic ...

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 ...

Columnist Patrick Moeschen was given an incorrect diagnosis of Becker muscular dystrophy, years before learning he actually ...

Advancing INLIGHT clinical study with novel, long-acting, muscle-sparing approach for obesity using Wave’s best-in-class GalNAc-siRNA ...

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

Equities researchers at Zacks Research cut their Q1 2025 earnings estimates for shares of Sarepta Therapeutics in a research ...