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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...

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Bibliografische gegevens
Gepubliceerd in:Am J Med Genet A
Hoofdauteurs: Viskochil, David, Clarke, Lorne A., Bay, Luisa, Keenan, Hillary, Muenzer, Joseph, Guffon, Nathalie
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: John Wiley & Sons, Inc. 2019
Onderwerpen:
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6899772/
https://ncbi.nlm.nih.gov/pubmed/31639289
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajmg.a.61378
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