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UnknownEvaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS).
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S-EPMC8557006 | biostudies-literature
UnknownLong-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series.
Not available
S-EPMC4158409 | biostudies-literature
UnknownFemale Patients With Mucopolysaccharidosis II (MPS II): Insights From the Hunter Outcome Survey.
Not available
S-EPMC12672032 | biostudies-literature
UnknownUnmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey.
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S-EPMC12211871 | biostudies-literature
UnknownClinical expert opinion on the role of elosulfase alfa in non-ambulatory individuals with Morquio A syndrome
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S-EPMC12859189 | biostudies-literature
UnknownEfficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study.
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S-EPMC4206772 | biostudies-literature
UnknownTen years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry.
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S-EPMC5414331 | biostudies-literature
UnknownRecommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance.
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S-EPMC6567385 | biostudies-literature
UnknownRecommendations for the management of MPS VI: systematic evidence- and consensus-based guidance.
Not available
S-EPMC6541999 | biostudies-literature
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