Biblio
High-Dose ERT, Rituximab, and Early HSCT in an Infant with Wolman's Disease. N Engl J Med. 2024;390(7):623-629.
. Longitudinal volumetric analysis of gray matter atrophy in metachromatic leukodystrophy. J Inherit Metab Dis. 2024.
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Metachromatic leukodystrophy and transplantation: remyelination, no cross-correction. Ann Clin Transl Neurol. 2020.
SALIVAL α-IDURONIDASE ACTIVITY AS A POTENTIAL NEW BIOMARKER FOR THE DIAGNOSIS AND MONITORING THE EFFECT OF THERAPY IN MUCOPOLYSACCHARIDOSIS I. Biol Blood Marrow Transplant. 2018.
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