Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

Although the year brought a steady stream of new treatments, tests, and cutting-edge science, experts worry about how ...

The ROAR trial tested the hypothesis that returning familial hypercholesterolemia-associated genetic results leads to ...

LEXINGTON, Mass. and AMSTERDAM, Jan. 09, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing ...

Co-founded by Jennifer Doudna and Fyodor Urnov, the company intends to simultaneously develop many gene editing treatments ...

The need to change regulations around gene-editing treatments was endorsed in November by the head of the US Food and Drug ...

Some parts of our bodies bounce back from injury in fairly short order. The outer protective layer of the eye—called the ...

An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

Expected rapid advances in CAR T and other cellular therapies may expand treatment options not only in cancer but also in ...

Medscape Medical News talked with researcher Stacie Dusetzina about the prospects for a single-payer program to help with the ...

Burkitt's lymphoma is a rare and aggressive blood cancer characterized by a translocation of the MYC gene. It occurs most ...