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Long-Term Outcomes of Early Enzyme Replacement Therapy for Mucopolysaccharidosis IV: Clinical Case Studies of Two Siblings

Enzyme replacement therapy (ERT) is one of the available therapies for mucopolysaccharidosis (MPS). This study presents a follow-up of two siblings with MPS IVA (Morquio A disease) that received ERT. Both siblings received weekly intravenous infusions of elosulfase alfa for 4.5 years. One sibling (p...

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Detalles Bibliográficos
Autores principales:	Barak, Sharon, Anikster, Yair, Sarouk, Ifat, Stern, Eve, Eisenstein, Etzyona, Yissar, Tamar, Sherr-Lurie, Nir, Raas-Rothschild, Annick, Guttman, Dafna
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2020
Materias:	Case Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7168314/ https://www.ncbi.nlm.nih.gov/pubmed/32079294 http://dx.doi.org/10.3390/diagnostics10020108

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