Enzyme replacement therapy for mucopolysaccharidosis VI from 8 weeks of age-a sibling control study
Author:
Publisher
Wiley
Subject
Genetics (clinical),Genetics
Link
http://onlinelibrary.wiley.com/wol1/doi/10.1111/j.1399-0004.2009.01324.x/fullpdf
Reference14 articles.
1. Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome);Swiedler;Am J Med Genet A,2005
2. Direct comparison of measures of endurance, mobility and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase;Harmatz;Pediatr,2005
3. Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study;Harmatz;J Pediatr,2006
4. Enzyme replacement therapy from birth in a feline model of mucopolysaccharidosis type VI.;Crawley;J Clin Invest,1997
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