Genzyme Corp.

Research activity includes Gaucher disease type 1 and Gaucher disease type 3, with attention to both systemic disease control and central nervous system involvement. The portfolio reflects interest in substrate reduction therapy, enzyme replacement therapy, and biomarker-based assessment in adult and pediatric populations.

• Eliglustat
• Imiglucerase
• Venglustat

Clinical interest extends to cerebrospinal fluid biomarkers, neurologic manifestations of Gaucher disease type 3, and treatment approaches spanning combination therapy and maintenance therapy.