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 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.

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 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.

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