Mass General Brigham: Gene Therapy Shows Long-Term Benefit for Patients With a Rare Pediatric Brain Disease
October 10, 2024
October 10, 2024
BOSTON, Massachusetts, Oct. 10 (TNSres) -- Mass General Brigham issued the following news release:
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Clinical trial of 32 patients with cerebral adrenoleukodystrophy found that after six years most remained free of major disabilities
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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. Resear . . .
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Clinical trial of 32 patients with cerebral adrenoleukodystrophy found that after six years most remained free of major disabilities
* * *
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. Resear . . .