Video: Gene Therapy
Spinal Muscular Atrophy (SMA) is caused by a gene mutation that leads to gradual, irreparable muscle damage with the majority of the children passing away before the age of two years. 10 to 15 years ago, there was no treatment. With the advent of gene therapies like those being developed at UVA’s Children Hospital, families have new hope and children a fresh start.
This video is the story of Isabelle, who received a diagnosis of SMA. Her family reached out to doctors at UVA Children’s Hospital who put the promise of gene therapy to work.
