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

Silvia Pontesilli,Cristina Baldoli,Pasquale Anthony Della Rosa,Alessandro Cattoni,Maria Ester Bernardo,Pamela Meregalli,Serena Gasperini,Serena Motta,Francesca Fumagalli,Francesca Tucci, Federica Baciga,Giulia Consiglieri,Francesco Canonico,Paola De Lorenzo,Luisa Chiapparini,Bernhard Gentner,Alessandro Aiuti,Andrea Biondi,Attilio Rovelli,Rossella Parini

The Journal of Pediatrics（2022）

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摘要

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.

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关键词

ERT and MPS,haematopoietic stem cell transplantation,HSCT,magnetic resonance,MPSI,MPSI-H,neuroradiology and MPS

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