Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System.
case_series · Level IV
Where this comes from
- Record sourced from PubMed, PMID 34547335.
- Also identified by DOI 10.1016/j.jpeds.2021.09.020.
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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.
Medical subject headings
- Brain
- Mucopolysaccharidosis I
- Spinal Cord