Long-term cognitive and somatic outcomes of enzyme replacement therapy in untransplanted Hurler syndrome

Mucopolysaccharidosis type I (MPS I) was added to the Recommended Uniform Screening Panel for newborn screening in 2016, highlighting recognition that early treatment of MPS I is critical to stem progressive, irreversible disease manifestations. Enzyme replacement therapy (ERT) is an approved treatm...

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Detalhes bibliográficos
Main Authors: Julie B. Eisengart, Jeanine Jarnes, Alia Ahmed, Igor Nestrasil, Richard Ziegler, Kathleen Delaney, Elsa Shapiro, Chester Whitley
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2017-12-01
Colecção:Molecular Genetics and Metabolism Reports
Assuntos:
Mucopolysaccharidosis
Enzyme replacement therapy
Cognitive decline
Newborn screening
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S2214426917301283
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