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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Sparad:
Bibliografiska uppgifter
I publikationen:Diagnostics (Basel)
Huvudupphovsmän: Barak, Sharon, Anikster, Yair, Sarouk, Ifat, Stern, Eve, Eisenstein, Etzyona, Yissar, Tamar, Sherr-Lurie, Nir, Raas-Rothschild, Annick, Guttman, Dafna
Materialtyp: Artigo
Språk:Inglês
Publicerad: MDPI 2020
Ämnen:
Case Report
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC7168314/
https://ncbi.nlm.nih.gov/pubmed/32079294
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/diagnostics10020108
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