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

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

source https://www.jpeds.com/article/S0022-3476(21)00894-5/fulltext?rss=yes

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