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Growth patterns for untreated individuals with MPS I: Report from the international MPS I registry
Mucopolysaccharidosis Type I (MPS I), caused by deficiency of α‐L‐iduronidase results in progressive, multisystemic disease with a broad phenotypic spectrum including patients with severe (Hurler syndrome) to attenuated (Hurler–Scheie and Scheie syndromes) disease. Disordered growth is common with e...
Autores principales: | Viskochil, David, Clarke, Lorne A., Bay, Luisa, Keenan, Hillary, Muenzer, Joseph, Guffon, Nathalie |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley & Sons, Inc.
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6899772/ https://www.ncbi.nlm.nih.gov/pubmed/31639289 http://dx.doi.org/10.1002/ajmg.a.61378 |
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