New spinal muscle atrophy treatment gains approval

Zolgensma is based on a delivery system discovered by a Penn gene therapy pioneer, marking a new milestone in treating the rare disease.

The U.S. Food and Drug Administration recently approved a gene therapy known as Zolgensma for spinal muscle atrophy, the most common inherited fatal disease in infants.

Genetic sequence

The treatment, which is based on a decade of work by a team under the leadership of James Wilson, director of the University of Pennsylvania’s Gene Therapy Program and Orphan Disease Center, and a professor of medicine and pediatrics in the Perelman School of Medicine, corrects a gene mutation that eventually impedes a young child’s ability to walk, eat, and breathe.

Read more at Penn Medicine News.