Thursday 6 June 2019

New gene therapy poised to transform care for spinal muscular atrophy

The University of Rochester Medical Center (URMC) 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 (SMA). The new treatment can be delivered within weeks of birth and clinical trials have shown that it dramatically changes the course of the disease.

* This article was originally published here