GAUCHER DISEASE TYPE 3
Clinical trials for GAUCHER DISEASE TYPE 3 explained in plain language.
Never miss a new study
Get alerted when new GAUCHER DISEASE TYPE 3 trials appear
Sign up with your email to follow new studies for GAUCHER DISEASE TYPE 3, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
New pill aims to tackle rare genetic disorder affecting brain and body
Disease control Not yet recruitingThis study tests an experimental drug called nizubaglustat (AZ-3102) in people aged 4 to 30 with Gaucher disease type 3, a rare genetic condition that can cause anemia, low platelets, an enlarged spleen, and neurological problems. Participants take the drug daily as a dissolvable…
Matched conditions: GAUCHER DISEASE TYPE 3
Phase: PHASE2 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jul 22, 2026 00:00 UTC
-
Gene therapy injection aims to control rare brain disease in children
Disease control Not yet recruitingThis early-stage trial tests a new gene therapy called VGN-R08b for children with type III Gaucher disease, a rare genetic disorder affecting the brain and body. The treatment is given as a single injection into the brain's fluid spaces. The study will monitor safety and measure …
Matched conditions: GAUCHER DISEASE TYPE 3
Phase: PHASE1, PHASE2 • Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 12:37 UTC