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Filters applied: Meta-Analysis, Randomized Controlled Trial. Clear all
Page 1
Galsulfase (Naglazyme) therapy in infants with mucopolysaccharidosis VI.
Harmatz PR, Garcia P, Guffon N, Randolph LM, Shediac R, Braunlin E, Lachman RS, Decker C. Harmatz PR, et al. J Inherit Metab Dis. 2014 Mar;37(2):277-87. doi: 10.1007/s10545-013-9654-7. Epub 2013 Oct 10. J Inherit Metab Dis. 2014. PMID: 24108527 Free PMC article. Clinical Trial.
RESULTS: Four infants (aged 3.3-12.7 months) participated in the study. Galsulfase was well tolerated at 1.0 and 2.0 mg/kg/week dose levels with no drug-related serious adverse events. ...Urinary GAG levels decreased with treatment. Early initiation of galsulfase ma …
RESULTS: Four infants (aged 3.3-12.7 months) participated in the study. Galsulfase was well tolerated at 1.0 and 2.0 mg/kg/week dose …
Efficacy of different treatment strategies in patients with mucopolysaccharidosis: a systematic review and network meta-analysis of randomized controlled trials.
Huang L, Wu J, Tang B, Wu J, Wei F, Li HQ, Li L, Wang X, Wang B, Wu W, Hong X. Huang L, et al. Orphanet J Rare Dis. 2025 May 2;20(1):211. doi: 10.1186/s13023-025-03735-y. Orphanet J Rare Dis. 2025. PMID: 40317013 Free PMC article.
In MPS VI, recombinant human arylsulfatase B (rhASB) and galsulfase (1.0 mg/kg/week) significantly reduced uGAG aggregation compared with the placebo group (-217, 95% CI[-258, -176]) and galsulfase (2.0 mg/kg/week) group (-286.5, 95% CI[-436.5, -136.5]), respectivel …
In MPS VI, recombinant human arylsulfatase B (rhASB) and galsulfase (1.0 mg/kg/week) significantly reduced uGAG aggregation compared …