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

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

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

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

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

UHealth and Holtz Children’s Hospital physicians are exploring a new gene therapy called ELEVIDYS to treat older Duchenne muscular dystrophy (DMD) patients. The therapy delivers a functional ...

Revidia Therapeutics Inc., a cardiac regenerative medicine company developing first-in-class small molecule drug therapies for heart injury, has announced that it has expanded its leadership team in ...

Capricor's biologics license application for deramiocel (CAP-1002) is draws largely on the Phase 2 HOPE-2 trial and its ...

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 ...

"By understanding how these proteins function differently in various tissues, we're one step closer to designing treatments that target the root causes of DMD." The study reveals that dystrophin's ...