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

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct ...

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

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

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

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

D Molecular Therapeutics (FDMT) offers attractive growth potential with strong cash reserves and promising late-stage gene ...

Stunning success in halting Huntington's disease with gene therapy offers new hope to those with the disorder—and potential ...

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

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

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

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