Journal article

Monitoring dose response of enzyme replacement therapy in feline mucopolysaccharidosis type VI by tandem mass spectrometry

A Crawley, SL Ramsay, S Byers, JJ Hopwood, PJ Meikle

Pediatric Research | NATURE PUBLISHING GROUP | Published : 2004

Abstract

Mucopolysaccharidosis type VI is an inherited disorder of glycosaminoglycan metabolism characterized by organomegaly, corneal clouding, and skeletal dysplasia. Recent developments in the use of tandem mass spectrometry to measure sulfated mono- and disaccharides have enabled us to perform noninvasive, biochemical monitoring during therapy regimes in mucopolysaccharidosis type VI cats in addition to established methods of disease evaluation. In this study, mucopolysaccharidosis type VI animals were given high-dose (20 mg/kg) enzyme replacement therapy for the first month after birth followed by low doses (1 mg/kg) for a further 2 mo and were compared with animals maintained on 1 mg/kg enzyme ..

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University of Melbourne Researchers