One of the demands raised during the protests was access to 'Elevidys', a one-time gene therapy treatment that has the ...

Florida leaders can enact a new law, the Right to Try for Individualized Treatments, that allows Floridians with rare ...

Edgewise Therapeutics plans to sell nearly 10 million shares of its common stock at $20.13, raising $200 million.

Barry Byrne, MD, PhD, Powell Gene Therapy Center at the University of Florida, discusses gene therapy considerations for pediatric patients and how newborn screening can influence outcomes for ...

Discover growth opportunities with Edgewise Therapeutics, Inc. in the hypertrophic cardiomyopathy market. Click for my EWTX ...

Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies for ...

Charley Seckler has found "humor in the madness" of rolling through life with a rare disease. He drops a blog post weekly, ...

Matthew B. Klein, M.D., the Chief Executive Officer of PTC Therapeutics, expressed disappointment at the EC’s decision but ...

Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

Muscular Dystrophy Association (MDA) has teamed up with Burn Boot Camp, a leading boutique fitness franchise, for the 9th annual national 'Be Their Muscle' philanthropic event. Throughout April, over ...

Trial achieved key objectives for safety, tolerability, pharmacokinetics and pharmacodynamicsClinical data from this trial, together with ...

New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...