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 ...

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

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 ...

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

Explore Solid Biosciences’ potential with promising SGT-003 trial results for DMD, strong financials, and market recovery ...

A few weeks after Sarepta Therapeutics confirmed a patient died after receiving its drug Elevidys, research published April 3 ...

The findings suggest that the body's own immune system may be undermining the treatment with gene therapy such as Elevidys ...