Ultrasonographic hip morphology in mucopolysaccharidosis type I Hurler after hematopoietic stem cell gene therapy

Hip morphology of children with mucopolysaccharidosis type I Hurler syndrome before and after hematopoietic stem cell gene therapy can be evaluated by available ultrasound techniques until a median age of 45 months.

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Publication Details

Journal
Journal of Children s Orthopaedics
Published
2025-08-11
DOI
https://doi.org/10.1177/18632521251349438
Primary Topic
Lysosomal Storage Disorders Research
Type
article
Field-Weighted Citation Impact
0.00
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article

Ultrasonographic hip morphology in mucopolysaccharidosis type I Hurler after hematopoietic stem cell gene therapy

Matilde Cossutta, Chiara Filisetti, Michele Colombo, Maurizio De Pellegrin et al.
Journal of Children s Orthopaedics
Lysosomal Storage Disorders Research
article

Ultrasonographic hip morphology in mucopolysaccharidosis type I Hurler after hematopoietic stem cell gene therapy

Matilde Cossutta, Chiara Filisetti, Michele Colombo, Maurizio De Pellegrin, Alessandro Aiuti, Francesca Tucci, Giulia Consiglieri, Maria Ester Bernardo
article en

Abstract

Hip morphology of children with mucopolysaccharidosis type I Hurler syndrome before and after hematopoietic stem cell gene therapy can be evaluated by available ultrasound techniques until a median age of 45 months.

Journal of Children s OrthopaedicsVol. 19(5)
University of Rome Tor Vergata (IT), Vita-Salute San Raffaele University (IT), San Raffaele University of Rome (IT), The San Raffaele Telethon Institute for Gene Therapy (IT)
Openalex Percentile: Top 74%
Lysosomal Storage Disorders Research
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