Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) ...

Untreated Spinal Muscular Atrophy leads to severe complications and declines in quality of life, emphasizing the need for ...

Capricor Therapeutics is seeking FDA approval for CAP-1002, targeting heart function in Duchenne Muscular Dystrophy patients.

New data demonstrated the benefits of Elevidys, a gene therapy for multiple Duchenne muscular dystrophy, in different age ...

Gene therapy could be the newest breakthrough for Duchenne muscular dystrophy. While it may not be a cure, it can give independence to boys diagnosed with DMD.

Multiple first-in-human trials showed functional and biomarker improvements in early data reported at ASGCT's annual meeting.

Data from the EMBARK trial suggest patients may have functional improvements taking the gene therapy between 8 and 9 years old.

Since Elevidys' accelerated approval in 2023, experts have been clamoring for more data, particularly in older and ...

Muscle weakness has the biggest impact on patients’ ability to walk, but other factors matter too, researchers found.

And for now, Logan and his family are reeling in the moments, one fish at a time.

Elevidys is a prescription gene therapy developed by Sarepta Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). It targets the root cause of the disease by delivering a shortened but ...

Quince Therapeutics advances Ataxia-Telangiectasia treatment with innovative AIDE platform, insider support, and NEAT Phase 3 ...