The purpose of the study is evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (MPS VI)
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
BioMarin Pharmaceutical Inc.
Novato, California, United States
12-minute walk test
Time frame: weeks 6, 12, 24, 48, 96 and 144
urinary glycoaminoglycan(uGAG)levels
Time frame: weeks 1, 4, 6, 8, 12
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