Department of Pediatrics

Lakewood family hopeful gene therapy treats ultra-rare disease

Written by 9News | December 21, 2023

A Lakewood couple learned both of their kids have SPG50, an ultra-rare genetic disease that affects cognitive and motor function. Though navigating SPG50 is far from what the family had planned, they're doing everything they can to help their kids, including gene therapy. Sarah Guistolisi, MD, neurology resident, explains more on treating this type of rare disease.