Treatment of mucopolysaccharidosis type II (Hunter syndrome) with idursulfase: the relevance of clinical trial end points.

Glamuzina, Emma

Treatment of mucopolysaccharidosis type II (Hunter syndrome) with idursulfase: the relevance of clinical trial end points. [electronic resource] - Journal of inherited metabolic disease Jun 2011 - 749-54 p. digital

Publication Type: Evaluation Study; Journal Article

1573-2665

10.1007/s10545-011-9280-1 doi


Child
Child, Preschool
Clinical Trials as Topic--methods
Clinical Trials, Phase II as Topic
Clinical Trials, Phase III as Topic
Endpoint Determination--methods
Enzyme Replacement Therapy
Humans
Iduronate Sulfatase--therapeutic use
Infant
Infant, Newborn
Male
Mucopolysaccharidosis II--diagnosis
Prognosis
Research Design
Retrospective Studies
Treatment Outcome