New Gene Therapy Poised to Transform Care for Spinal Muscular Atrophy

The University of Rochester Medical Center has been tapped as one of the first institutions in the U.S. to offer a new gene replacement therapy to treat spinal muscular atrophy. The new treatment, which is being marketed by Novartis under the brand name Zolgensma, can be delivered within weeks of birth and clinical trials have shown that it dramatically changes the course of the disease.
Source: University of Rochester Medical Center Press Releases - Category: Universities & Medical Training Authors: Source Type: news