Treatment introduces an expansion of the scope of gene therapies that might lead to similar treatments for neurodegenerative ...

After three patients died, the maker of an investigational gene therapy stopped shipments of the drug after it refused to ...

A global clinical trial for a new Huntington’s disease treatment has posted positive results today, announced by trial ...

AMT-130 gene therapy slowed Huntington’s progression by 75%, offering the first evidence of a disease-modifying treatment.

EXTON, PA, Aug. 14, 2025 (GLOBE NEWSWIRE) -- As the first wave of gene therapies for transfusion-dependent β-thalassemia (TDT) continues to roll out in the U.S., a new conversation is emerging within ...

Sarepta Therapeutics’ report of a second fatality this year in a patient dosed with its Duchenne muscular dystrophy gene therapy raised the prospect it might have to pull the product from the market.

Pediatric participants demonstrated large gains in cone-mediated vision; therapy remains well tolerated with no ocular serious adverse events or ...

This article is part of Nature Outlook: Skin, an editorially independent supplement produced with financial support from LEO Pharma. About this content. KaDee Troop is the mother of seven adopted ...

Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid ...

Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...