Conditions: Mucopolysaccharidosis VI
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 39
Healthy volunteers: no

Brief summary:
The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk test at Week 24 compared with baseline.

Detailed description:

Arms:
arm 1: None arm 2: None

Interventions: intervention 1: Placebo intervention 2: N-acetylgalactosamine 4-sulfatase
Intervention descriptions: intervention 1: None intervention 2: None

Protocol document: