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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...
Autores principales: | Polgreen, Lynda E., Bay, Luisa, Clarke, Lorne A., Guffon, Nathalie, Jones, Simon A., Muenzer, Joseph, Flores, Ana Lorena, Wilson, Kathryn, Viskochil, David |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley & Sons, Inc.
2022
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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 |
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