Clinical Drug Experience Knowledgebase

Criteria

Inclusion Criteria:

Patient consent
Patient must be five years of age or older
Patient must have documented diagnosis of MPS VI, confirmed at screening by measurable clinical signs and symptoms of MPS VI
Leukocyte ASB enzyme activity level less than 20% of the normal range
Clinical evidence of significant MPS VI disease that provides adequate opportunity to achieve quantitative, short-term therapeutic benefit in three or more of the following parameters: endurance (as measured by a six-minute walk test), forced vital capacity (as measured by spirometry), joint range of motion, urinary glycosaminoglycans, and hepatomegaly.
Ability to perform all protocol tests
Ability to stand independently for six minutes
Sexually active subjects must agree to use an adequate form of contraception

Exclusion Criteria:

History of bone marrow transplantation
Pregnant or lactating patient
Use of an investigational drug or device within 30 days prior to study participation.
A medical condition, serious intercurrent illness, or other extenuating circumstances that may significantly decrease study compliance including prescribed follow-up
Known hypersensitivity to rhASB or to components of the study drug
History of cancer (except low grade and fully resolved skin malignancy)