Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome): defining and measuring functional impacts in pediatric patients

被引：0
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作者：
Beth Leiro
Dawn Phillips
Melanie Duiker
Paul Harmatz
Sharon Charles
机构：
[1] Phillips Consulting,
[2] Paradigm Biopharmaceuticals Limited,undefined
[3] UCSF Benioff Children’s Hospital,undefined
来源：
Orphanet Journal of Rare Diseases | / 16卷
关键词：
Clinical Outcome Assessment; Focus Groups; Mucopolysaccharidosis VI; Pain; Patient Reported Outcome Measures; Pediatrics;
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