News and Trends 28 Mar 2019
Gene Therapy Exceeds Expectations in Treating Children’s Neurological Disease
A gene therapy developed by the UK company Orchard Therapeutics has greatly improved the motor symptoms of children with an incurable neurological disease. The 20 children in the phase II trial suffered from metachromatic leukodystrophy, a rare genetic disease caused by the mutation of a protein that breaks down sulfur-containing molecules in cells. This leads […]