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.

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

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.

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

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

Sarepta Therapeutics, Inc. , the leader in precision genetic medicine for rare diseases, today shared updates from its clinical programs focused on limb-girdle muscular dystrophy (LGMD) subtypes 2C/R5 ...

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

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

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

Upon approval, vamorolone would be the first and only treatment option indicated for patients diagnosed with Duchenne muscular dystrophy in Canada The Canadian regulatory submission of vamorolone ...

HENLEY MP Freddie van Mierlo has called on a hospital trust to apologise for “misleading” the family of a boy with a muscle-wasting condition. Ben Clarke, 11, a pupil at Gillotts School in Henley, has ...