Tag Archive for: Duchenne muscular dystrophy (DMD)

Sarepta’s Elevidys, approved Thursday as the first gene therapy for Duchenne muscular dystrophy, has been priced at $3.2 million per patient, making it one of the world’s most expensive medicines.

Entrada Therapeutics said it received a clinical hold notice from the regulatory agency but no reason was provided. “We are eager to resolve this hold and continue down the treatment development pathway,” President and CEO Dipal Doshi said.

The FDA removed the clinical hold on Sarepta Therapeutics’ investigational Duchenne muscular dystrophy (DMD) therapy Tuesday after the company agreed to adjust its clinical trial protocols to include expanded monitoring of urine biomarkers. 

One of the tenets of “personalized medicine” is that medications will be tailored to individuals based on their personalized genetics. Last year, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research approved 50 drugs. A new study found that 33, or 66% of them, were supported with genomic data.

Research scientists from the School of Biomedical Engineering at the University of British Columbia have found that a colony-stimulating factor 1 receptor (CSF1R) inhibitor, best known as a cancer drug, may also be a potential treatment for Duchenne muscular dystrophy (DMD), a severe genetic disorder afflicting the muscles.

Sarepta Therapeutics announced Thursday that the U.S. Food and Drug Administration has put the Phase II trial of its Duchenne muscular dystrophy (DMD) candidate SRP-5051 (vesleteplirsen) on clinical hold following a serious safety signal.