3 results
Approved WMOCompleted
The aim of this study is to collect data in a structured way in order to explore the tolerability, safety and efficacy of paliperiode ER with treatment schedules which will be used in future clinical practice.
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.
Approved WMORecruiting
Primairy objectives 1. To determine the presence and extent of myocardial microcalcification and myocardial denervation in ATTR-CM;2. To quantify the burden of myocardial microcalcification and myocardial denervation in ATTR-CM;3. To assess whether…