Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series

Title
Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series
Authors
Keywords
Enzyme Replacement Therapy, Allergic Conjunctivitis, Health Assessment Questionnaire Disability Index, Somatic Sign, Child Health Assessment Questionnaire
Journal
JOURNAL OF INHERITED METABOLIC DISEASE
Volume 37, Issue 5, Pages 823-829
Publisher
Springer Nature
Online
2014-03-04
DOI
10.1007/s10545-014-9686-7

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