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.
Editoriale, introduzione, contributo a forum/dibattito
ERT and MPS; haematopoietic stem cell transplantation; HSCT; magnetic resonance; MPSI; MPSI-H; neuroradiology and MPS;
English
20-set-2021
2022
240
January 2022
297
301.e5
none
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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