Enzyme replacement therapy for heart health in Gaucher type 3

This study tests whether a new drug called VGN-R08b can safely help children with Type III Gaucher disease—a rare genetic disorder affecting the brain and body. The drug is being tested in children aged 2–17 who have already tried other treatments but still have neurological symptoms, particularly eye movement problems.

This study looks at MRI-based lab measurements (similar to a special type of scan) to find markers that reflect health in people with type 3 Gaucher disease. It may help researchers track the disease over time and improve future treatments.

This trial tests an experimental drug called nizubaglustat (AZ-3102) for people with Gaucher disease type 3. It aims to see if the drug can improve symptoms like anemia, low platelets, and enlarged spleen, which are common in this condition.

This study looks at people with Gaucher disease (types I, II, or III) to better “group” patients for understanding and future research. If you (or your child) have confirmed Gaucher disease, you may be asked to share information and follow a study schedule.

This trial provides access to venglustat, an experimental oral medication for people with type 3 Gaucher disease that affects the brain and nervous system. It is designed for those who are already on a stable dose of enzyme replacement therapy (ERT) and are looking for additional treatment options.

This study checks whether certain heart-related blood tests and heart scans can help monitor people with Fabry disease. It may be useful for both people who have never received enzyme replacement therapy and those who have.