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 ...

A new study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein, dystrobrevin, offering new pathways for understanding and ...

A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein, dystrobrevin, offering new pathways for ...

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

Wave Life Sciences shows promising data in genetic mutation treatments, with GSK collaborations and strong financial footing.

New study reveals crucial protein interactions in Duchenne Muscular Dystrophy, opening doors to more precise and effective ...

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

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 ...

A new study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its ...

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