The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in patients diagnosed with MPS VI.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
BioMarin Pharmaceutical Inc.
Novato, California, United States
Lysosomal storage disease
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.