2 clinical trials found.
-
An Observational Longitudinal Prospective Long-Term Registry of Patients With Hypophosphatasia
This multinational, multicenter, observational, prospective, long-term registry is designed to collect data on epidemiology, hypophosphatasia (HPP) history, clinical course, symptoms (including systemic aspects of disease ... -
Gaucher Disease Outcome Survey (GOS)
Gaucher disease is one of several rare lysosomal storage disorders, affects 10,000-30,000 patients worldwide, and is classified into three clinical subtypes. Type 1 ...