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

A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin ...

An experimental gene therapy was found to slow the progression of Huntington’s disease in a “pivotal” study, according to ...

A one-time gene therapy called AMT-130 helped slow the progression of Huntington's disease by 75%, paving the way for the ...

A pioneering gene therapy has successfully slowed the progression of Huntington’s disease by 75% in patients after three ...

Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed.

The FDA has granted Fast Track designation to SAR446268 for the treatment of non-congenital myotonic dystrophy type 1.

A company called uniQure has announced promising results from a trial of a new gene therapy for Huntington's disease. The ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the ...

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

Jesse Gelsinger died after receiving a gene therapy treatment to treat a liver disease. The death sparked an investigation ...

"On the basis of these results, it seems likely AMT-130 will be the first licensed treatment to slow Huntington's disease, ...