We developed a brain and spine magnetic resonance scoring system based on a magnetic resonance assessment of 9 patients with mucopolysaccharidosis type I-Hurler who underwent hematopoietic stem-cell transplantation. The score is reliable and correlates with long-term clinical and cognitive outcome in patients with mucopolysaccharidosis type I-Hurler.

Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System

Bernardo M. E.;Consiglieri G.;Aiuti A.;
2022-01-01

Abstract

We developed a brain and spine magnetic resonance scoring system based on a magnetic resonance assessment of 9 patients with mucopolysaccharidosis type I-Hurler who underwent hematopoietic stem-cell transplantation. The score is reliable and correlates with long-term clinical and cognitive outcome in patients with mucopolysaccharidosis type I-Hurler.
2022
20-set-2021
Inglese
Elsevier Inc.
240
297
301
5
Pubblicato
Esperti anonimi
Internazionale
ERT and MPS
haematopoietic stem cell transplantation
HSCT
magnetic resonance
MPSI
MPSI-H
neuroradiology and MPS
No
none
20
info:eu-repo/semantics/article
262
Pontesilli, S.; Baldoli, C.; Rosa, P. A. D.; Cattoni, A.; Bernardo, M. E.; Meregalli, P.; Gasperini, S.; Motta, S.; Fumagalli, F.; Tucci, F.; Baciga, ...espandi
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/20.500.11768/120625
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