Gene Therapy With Research Origins In Greater Philadelphia Approved By FDA to Treat Spinal Muscular Atrophy

The FDA approved of a gene therapy called Zolgensma that was developed by a team of researchers at the University of Pennsylvania to correct the genes that cause spinal muscle atrophy (SMA).

SMA is caused by a mutation of a specific gene, SMN1, that affects nerve cells in the spinal cord. Zolgensma is a one-time treatment that provides a functional, non-mutated copy of SMN1 to restore production.

Article by Penn Medicine