In a world first, a bespoke gene-editing therapy benefitted one child. Now reseachers plan to launch a clinical trial of the ...
Pivotal results from uniQure’s gene therapy for Huntington’s disease have brought new light to patients who have known only ...
A top US regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare ...
Shortly after taking reins at BioMarin, CEO Alexander Hardy laid out three paths the company could take for its struggling ...
In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians ...
Columnist Jennifer Lynne concludes a series on Domenic Catrine, whose life has improved after receiving gene therapy to treat hemophilia B.
University of Bristol researchers have discovered a VEGF-C gene therapy that protects the kidneys in people with type 1 ...
Just as Adverum Biotechnologies' cash reserves were running dry, Eli Lilly has swooped in | Just as Adverum’s cash reserves ...
Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...
Some of the most expensive drugs currently in use are gene therapies to treat specific diseases, and their high cost limits ...
The U.S. Food and Drug Administration has placed a clinical hold on Intellia Therapeutics' two late-stage trials testing an ...
Cleveland Clinic Children’s researchers have uncovered a genetic link to osteosarcoma, the most common bone cancer in ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback