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Additional file 1 of Clinical and event-based outcomes of patients with mucopolysaccharidosis VI receiving enzyme replacement therapy in Turkey: a case series

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posted on 2021-10-20, 03:25 authored by Aslı İnci, İlyas Okur, Leyla Tümer, Gürsel Biberoğlu, Murat Öktem, Fatih Ezgü
Additional file 1. S1. Urinary glycosaminoglycan (uGAG) levels over time. S2. Height and mucopolysaccharidosis VI-specific height percentile (based on Quartel et al., 2015 [1]) before treatment initiation and follow-up (boys and girls). S3. Cardiac echocardiography findings at initiation of enzyme replacement therapy (baseline) and at last follow-up. S4. Cranial and spinal magnetic resonance (MRI) outcomes at initiation of enzyme replacement therapy at baseline (BL) and follow-up (FU). S5. Urinary incontinence at initiation of enzyme replacement therapy (baseline) and at last follow-up. S6. Cumulative number of events by age before and after initiation of ERT. Individual patient data. S7. Summary of even-based outcomes.

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biomarin pharmaceutical

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    Orphanet Journal of Rare Diseases

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