Article
Growth patterns for untreated individuals with MPS I: Report from the international MPS I registry.
American journal of medical genetics. Part A - 1 Dec 2019
Viskochil David, Clarke Lorne A, Bay Luisa, Keenan Hillary, Muenzer Joseph, Guffon Nathalie
Abstract excerpt
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 either phenotype. The study objectives were to construct sex- and age-specific estimated length/height and head...
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