Type 3 Gaucher disease

Clinical trials for Type 3 Gaucher disease explained in plain language.

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New drug offers hope for brain symptoms in rare gaucher disease

Disease control Expanded access

This program provides early access to the experimental drug venglustat for children and adults with Type 3 Gaucher disease who have brain-related symptoms. Participants must be at least 12 years old, weigh at least 15 kg, and be stable on standard enzyme replacement therapy. The …

Matched conditions: TYPE 3 GAUCHER DISEASE

Sponsor: Sanofi • Aim: Disease control

Last updated Jul 02, 2026 00:00 UTC

Type 3 Gaucher disease

Get alerted when new Type 3 Gaucher disease trials appear

New drug offers hope for brain symptoms in rare gaucher disease