2 results
Approved WMORecruiting
1. To assess serum calcification propensity (T50) in SSc patients compared with healthy age and gender-matched controls; 2. To assess serum T50 in SSc patients with overt calcinosis cutis as compared to age and gender-matched SSc patients without; 3…
Approved WMOWill not start
The purpose of this study is to evaluate the possible risks and efficacy (improvement of disease) with an experimental oral study drug named eliglustat in pediatric patients from 2 to 18 years with Gaucher disease.