Extrait

Part 1: -Evaluate central nervous system (CNS) biomarkers in Gaucher disease (GD) type 3 (GD3) that distinguish GD3 from Gaucher disease type 1 (GD1). -Screen adult GD3 patients who qualify for treatment with GZ/SAR402671 in Part 2. Part 2: -Evaluate the safety and tolerability of GZ/SAR402671 in adult GD3 patients. -Evaluate the change in cerebrospinal fluid (CSF) central nervous System (CNS) biomarkers from adult GD3 patients receiving GZ/SAR402671.

Critère d'inclusion

• Gaucher Disease

Liens

• euctr