Optimizing Treatment for Cerebral Adrenoleukodystrophy in the Era of Gene Therapy

Adrenoleukodystrophy is a peroxisomal metabolic disorder that can be manifested by rapidly progressive cerebral demyelination (known as cerebral adrenoleukodystrophy) in affected boys and men. Untreated cerebral adrenoleukodystrophy causes severe disability or death approximately 2 years after its…

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