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

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

Santhera Pharmaceuticals’ Agamree (vamorolone) has been accepted by the Scottish Medicines Consortium (SMC) to treat Duchenne ...

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 ...

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 ...

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

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

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

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

Hallmark, a leading healthcare workforce management technology company, today announced the launch of the Hallmark Changemakers program, an initiative to recognize healthcare leaders using innovative ...

The 9% adjusted figure reflects the ability of the drug to penetrate into muscle tissue at high concentrations, said Wave in a letter to the DMD patient community, which reads: "Dystrophin was ...