Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis VI.

ABSTRACT:

Objective

To evaluate the efficacy and safety of two dose levels of galsulfase (Naglazyme®) in infants with MPS VI.

Study design

This was a phase 4, multicenter, multinational, open-label, two-dose level study. Subjects were randomized 1:1 to receive weekly infusions of 1.0 or 2.0 mg/kg of galsulfase for a minimum of 52 weeks. Progression of skeletal dysplasia was determined by monitoring physical appearance, radiographic changes, and growth. Urinary glycosaminoglycan (GAG) levels, gross and fine motor function, cardiac function, vision, hearing, and health resource utilization were evaluated. Safety assessments were performed.

Results

Four infants (aged 3.3-12.7 months) participated in the study. Galsulfase was well tolerated at 1.0 and 2.0 mg/kg/week dose levels wit

SUBMITTER: Harmatz PR 

PROVIDER: S-EPMC3976509 | biostudies-literature | 2014 Mar

REPOSITORIES: biostudies-literature