Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System
Articolo
Data di Pubblicazione:
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.
Tipologia CRIS:
1.1 Articolo in rivista
Elenco autori:
Pontesilli, S.; Baldoli, C.; Rosa, P. A. D.; Cattoni, A.; Bernardo, M. E.; Meregalli, P.; Gasperini, S.; Motta, S.; Fumagalli, F.; Tucci, F.; Baciga, F.; Consiglieri, G.; Canonico, F.; De Lorenzo, P.; Chiapparini, L.; Gentner, B.; Aiuti, A.; Biondi, A.; Rovelli, A.; Parini, R.
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