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-l

Article ID	Journal	Published Year	Pages	File Type
4169379	The Journal of Pediatrics	2006	13 Pages	PDF

Abstract

rhASB significantly improves endurance, reduces GAG, and has an acceptable safety profile.

Keywords

FVC GAG MVV IAR infusion-associated reaction enzyme replacement therapy Maximum voluntary ventilation Range of motion ROM forced vital capacity adverse event ASB Mucopolysaccharidosis MPs ERT Glycosaminoglycan

Related Topics

Health Sciences Medicine and Dentistry Perinatology, Pediatrics and Child Health

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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-l

Authors

Paul MD, Roberto MD, PhD, Ida MD, Nathalie MD, Elisa LeÃ£o MD, M. Clara Sá PhD, J. Edmond MD, Michael MD, Laila MD, Maurizio MD, Zi-Fan ScD, Janet PhD, Kenneth I. MD, Mary S. MS, Ann M. MD, Emil MD, PhD, Stuart J. MD, PhD,