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

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

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

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

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

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

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

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

Results from a three-year trial suggest an experimental gene therapy for Huntington's disease can slow the progression of the ...

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

A one-time experimental treatment shows unprecedented success in slowing Huntington's disease, according to UCL researchers.

In a trial of people with early Huntington's, the newly developed therapy slowed the progression of symptoms by 75%.