Haematopoietic stem cell transplantation does not retard disease progression in the psycho‐cognitive variant of late‐onset metachromatic leukodystrophy

Abstract excerpt

Haematopoietic stem cell transplantation has an unproven role in the management of late-onset metachromatic leukodystrophy: theoretically justified through the engraftment of enzyme-replete haematopoietic progenitors and restoration of capacity for sulphatide catabolism in neural tissue through enzyme recapture, the long-term outcome is unknown. The rarity of the psycho-cognitive variant and slow progression of...

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