Gene therapy shows long-term benefit for patients with a rare pediatric brain disease
Cerebral adrenoleukodystrophy (CALD) is a rare progressive, genetic brain disease that primarily presents in young boys, causing loss of neurological function and ultimately leading to early death. Researchers from Massachusetts General Hospital, a founding member of the Mass General Brigham healthcare system, Boston Children’s Hospital, and collaborators have shown that six years after treatment with the first gene therapy approved for CALD, 94 percent of patients have had no decline in neurological functioning, with over 80 percent remaining free of major disability. Findings, published in two articles in the New England Journal of Medicine, describe long-term outcomes ...













