Managed Access Program of Venglustat in Pediatric and Adult Patients With Type 3 Gaucher Disease (GD3).

Summary

The objective of this program is to provide early access to venglustat for certain patients with Gaucher disease type 3 in response to unsolicited requests from healthcare providers. No approved therapies for the CNS manifestations of the disease are currently available. The program will provide access to venglustat prior to registration and the availability of commercial product (including reimbursement where applicable) in the country of the patient.

Timeline

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Completion

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Drugs

Indications