Gene Therapy for Pompe Disease Effective in Mice, Poised for Human Trials
Single dose of gene therapy could augment or replace frequent enzyme infusions DURHAM, N.C. January 26, 2017 – After decades investigating a rare, life-threatening condition that cripples the muscles, Duke Health researchers have developed a gene therapy they hope could enhance or even replace the only FDA-approved treatment currently available to patients. The gene therapy, […]