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

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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Bibliographic Details
Main Authors: Sharon Barak, Yair Anikster, Ifat Sarouk, Eve Stern, Etzyona Eisenstein, Tamar Yissar, Nir Sherr-Lurie, Annick Raas-Rothschild, Dafna Guttman
Format: Article
Language:English
Published: MDPI AG 2020-02-01
Series:Diagnostics
Subjects:
case report
enzyme replacement therapy
glycosaminoglycan
deficient n-acetylgalactosamine 6-sulfatase
mucopolysaccharidosis
morquio syndrome
Online Access:https://www.mdpi.com/2075-4418/10/2/108

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