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

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

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

The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin”) in skeletal muscle.

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

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

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

The results suggest that prolonging ambulation may not adversely impact cardiac function in adulthood for patients with ...

After hours: January 14 at 6:28:07 PM EST Loading Chart for SRPT ...

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

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 ...

Q'Apel Medical (Q'Apel), a privately held medical device company focused on revolutionizing neurovascular interventions, ...