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Growth in individuals with attenuated mucopolysaccharidosis type I during untreated and treated periods: Data from the MPS I registry

Mucopolysaccharidosis Type I (MPS I) is caused by deficiency of α‐L‐iduronidase. Short stature and growth deceleration are common in individuals with the attenuated MPS I phenotype. Study objectives were to assess growth in individuals with attenuated MPS I enrolled in The MPS I Registry while untre...

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Detalles Bibliográficos
Autores principales: Polgreen, Lynda E., Bay, Luisa, Clarke, Lorne A., Guffon, Nathalie, Jones, Simon A., Muenzer, Joseph, Flores, Ana Lorena, Wilson, Kathryn, Viskochil, David
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9545955/
https://www.ncbi.nlm.nih.gov/pubmed/35869927
http://dx.doi.org/10.1002/ajmg.a.62910