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Clinical and Event-Based Outcomes of Patients With Mucopolysaccharidosis VI Receiving Enzyme Replacement Therapy in Turkey: A Case Series

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source: Orphanet journal of rare diseases

year: 2021

authors: İnci A,Okur İ,Tümer L,Biberoğlu G,Öktem M,Ezgü F

summary/abstract:

The objective of this study was to describe clinical manifestations and events of patients with mucopolysaccharidosis (MPS) VI in Turkey who are treated with galsulfase enzyme replacement therapy (ERT). Clinical data of 14 children with MPS VI who were followed up at the Department of Pediatrics of the Gazi University Faculty of Medicine in Ankara, Turkey were retrospectively collected from the patients’ medical records. Patients were selected based on availability of a pre-ERT baseline and follow-up clinical data for a similar period of time (1.9-3.2 years). Event data (occurrence of acute clinical events, onset of chronic events, surgeries) collected during hospital visits and telemedicine were available for up to 10 years after initiation of ERT (2.5-10 years).

organization: Department of Pediatrics, Division of Pediatric Metabolic Diseases, Faculty of Medicine, Gazi University, Mevlana Bulvarı No 29, Emniyet Mahallesi, Yenimahalle, Ankara, 06560, Turkey.

DOI: 10.1186/s13023-021-02060-4

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