Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?
PLOS: DNA Science
OCTOBER 12, 2023
The package insert for the drug, Elevidys , warns of adverse effects of acute liver injury and inflammation around the heart and muscles. million DNA bases. The DMD gene therapy delivers a shortened version of the dystrophin gene, just 4,558 DNA bases. He was treated on a Monday and died that Friday, as his organs shut down.
Let's personalize your content