News

An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...
The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...
Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...
REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...
Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.
The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...
The Duchenne muscular dystrophy market is witnessing steady growth driven by advancements in gene therapies, exon-skipping drugs, and corticosteroid alternatives. Increasing diagnosis rates, rising ...