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Figure 3 | Orphanet Journal of Rare Diseases

Figure 3

From: Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease

Figure 3

Clinical effects of enzyme replacement therapy (ERT) with idursulfase (0.5 mg/kg weekly) or placebo in patients with mucopolysaccharidosis type II (MPS II). (a) Mean (± SE) change from baseline in distance walked in 6-minute walk test; (b) mean (± SE) change from baseline in percentage of predicted forced vital capacity; (c) mean (± SE) change from baseline in absolute forced vital capacity; (d) mean (± SE) change from baseline in concentration of urine glycosaminoglycans; (e) mean (± SD) growth velocity before and during enzyme replacement therapy; (f) incidence of infusion reactions during treatment with idursulfase. Absolute forced vital capacity is a better measure of respiratory function than percentage of predicted forced vital capacity, as the latter assumes both normal growth and height, which does not apply to patients with MPS II. (a-d) adapted from [34] with permission; (e) reproduced from [37] with kind permission from Springer Science & Business Media; (f) reproduced from [35] with permission. *p = 0.0131; **p = 0.011; ***p < 0.0001, compared with placebo based on analysis of covariance. SD = standard deviation. SE = standard error. ANCOVA = analysis of covariance. FVC = forced vital capacity. ERT = enzyme replacement therapy.

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