Rare diseases
Investigational molecules
Locations
450 Water Street, 02141-2288, United States
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.
Clinical interest extends to cerebrospinal fluid biomarkers, neurologic manifestations of Gaucher disease type 3, and treatment approaches spanning combination therapy and maintenance therapy.
Genzyme Corp. runs clinical trials in conditions such as Gaucher's disease type III, Gaucher's disease type I, Haemophilia, Primary progressive multiple sclerosis, among others.
Clinical trials registered by this sponsor – none is enrolling right now.
Study on Tolebrutinib for Patients with Primary Progressive Multiple Sclerosis
AustriaStudy of Eliglustat alone or with imiglucerase in children and adolescents aged 2-17 years with Gaucher disease type 1 and type 3
FranceStudy of Long-term Safety and Efficacy of Fitusiran in Patients with Hemophilia A or B with or without Inhibitory Antibodies to Factor VIII or IX
DenmarkStudy of Fitusiran for Preventing Bleeding in Boys Aged 1 to 12 with Hemophilia A or B
ItalyStudy of Venglustat and Imiglucerase for Adult Patients with Gaucher Disease TYPE 3
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