Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

Despite being known for more than 150 years, Duchenne muscular dystrophy (DMD) remains an untreatable disease affecting approximately 1 of every 3,500-5,000 males. Muscles in patients express no or ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

HENLEY MP Freddie van Mierlo has called on a hospital trust to apologise for “misleading” the family of a boy with a ...

Keros Therapeutics halted phase 2 of TROPOS, but KER-065 shows promise in DMD and financial stability ensures growth ...

None of the eteplirsen-treated patients reached a left ventricular ejection fraction below 50% compared with 22.1% of ...

Learn more about whether Alvotech or Sarepta Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

Health Canada granted priority review to an application seeking approval of vamorolone, marketed in the U.S. as Agamree, for ...

Lexeo Therapeutics’ gene therapy provides a healthy version of the frataxin gene, which is critical for energy production in ...

CureDuchenne, led by CEO Debra Miller, is at the forefront of research for Duchenne muscular dystrophy, which is a rare and fatal neuromuscular disease.