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-Reference-Cited by-同舟云学术

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

Published:2006-04 Issue:4 Volume:148 Page:533-539.e6
ISSN:0022-3476
Container-title:The Journal of Pediatrics
language:en
Short-container-title:The Journal of Pediatrics

Author:

Harmatz Paul,Giugliani Roberto,Schwartz Ida,Guffon Nathalie,Teles Elisa Leão,Miranda M. Clara Sá,Wraith J. Edmond,Beck Michael,Arash Laila,Scarpa Maurizio,Yu Zi-Fan,Wittes Janet,Berger Kenneth I.,Newman Mary S.,Lowe Ann M.,Kakkis Emil,Swiedler Stuart J.

Publisher

Elsevier BV

Subject

Pediatrics, Perinatology and Child Health

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4. 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

5. Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome);Harmatz;J Pediatr,2004

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