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.

Pontesilli, S., Baldoli, C., Della Rosa, P., Cattoni, A., Bernardo, M., Meregalli, P., et al. (2022). Evidence of treatment benefits in patients with MPSI-Hurler in long-term follow up using a new MRI scoring system. THE JOURNAL OF PEDIATRICS, 240(January 2022), 297-301.e5 [10.1016/j.jpeds.2021.09.020].

Evidence of treatment benefits in patients with MPSI-Hurler in long-term follow up using a new MRI scoring system

Cattoni, Alessandro;Motta, Serena;Biondi, Andrea;
2022

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.
Articolo in rivista - Contributo a Forum/Dibattito, Introduzione
ERT and MPS; haematopoietic stem cell transplantation; HSCT; magnetic resonance; MPSI; MPSI-H; neuroradiology and MPS;
English
2022
297
301.e5
5
Pontesilli, S., Baldoli, C., Della Rosa, P., Cattoni, A., Bernardo, M., Meregalli, P., et al. (2022). Evidence of treatment benefits in patients with MPSI-Hurler in long-term follow up using a new MRI scoring system. THE JOURNAL OF PEDIATRICS, 240(January 2022), 297-301.e5 [10.1016/j.jpeds.2021.09.020].
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10281/328924
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