This is the first successful gene therapy treatment to halt a fatal brain disease. (clinicalresearchforum.org)
It was one of only a handful of successful gene therapy trials in people. (newscientist.com)
Washington, D.C. — April 18, 2018 — The Clinical Research (CR) Forum, a non-profit membership association of top clinical research experts and thought leaders from the nation's leading academic health centers, today awarded its most prestigious honor to a Massachusetts General Hospital research team for its discovery of the first successful gene therapy treatment for a fatal brain disease, cerebral adrenoleukodystrophy (ALD). (clinicalresearchforum.org)