Effectiveness of early initiation of idursulfase in infants and very young children with mucopolysaccharidosis II: a retrospective chart review in siblings
Infants treated earliest for Hunter syndrome showed better growth and fewer organ problems than older siblings who started treatment later.
INTRODUCTION: Enzyme replacement therapy (ERT) with intravenous idursulfase is the standard of care for patients with mucopolysaccharidosis II (MPS II; Hunter syndrome). GLMM analyses showed that, compared with their older siblings, patients who initiated ERT aged ≤12 months had better clinical outcomes for height-for-age Z-score, liver, cardiac, and musculoskeletal manifestations.
What the study was
- Study design
- Observational/Cohort Study
- Population
- patients with mucopolysaccharidosis II
- Sample size
- 38
- Category
- Drug Development
- Maturity
- Validated
- Journal
- Molecular genetics and metabolism reports
Why it surfaced
high triage score
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