Growth Charts for Individuals with Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome).

ABSTRACT:

Background

The skeletal phenotype of mucopolysaccharidosis VI (MPS VI) is characterized by short stature and growth failure.

Objective

The purpose of this study was to construct reference growth curves for MPS VI patients with rapidly and slowly progressive disease.

Methods

We pooled cross-sectional and longitudinal height for age data from galsulfase (Naglazyme(®), BioMarin Pharmaceutical Inc.), treatment naïve patients (n = 269) who participated in various MPS VI studies, including galsulfase clinical trials and their extension programs, the MPS VI clinical surveillance program (CSP), and the MPS VI survey and resurvey studies, to construct growth charts for the MPS VI population. There were 229 patients included in this study, of which data from 207 patients ≤25 ye

SUBMITTER: Quartel A 

PROVIDER: S-EPMC4361922 | biostudies-literature | 2015

REPOSITORIES: biostudies-literature