GAUCHER DISEASE TYPE 3

Clinical trials for GAUCHER DISEASE TYPE 3 explained in plain language.

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Trials to join now! 2 Not yet recruiting 1 Terminated 1

Gene therapy injection aims to control rare brain disease in kids

Disease control Not yet recruiting

This early-stage study tests a new gene therapy called VGN-R08b, given as a single injection into the brain fluid, for children aged 2 to 17 with Type III Gaucher's disease. The goal is to see if it is safe and can improve enzyme activity related to the disease. Only 12 participa…

Matched conditions: GAUCHER DISEASE TYPE 3

Phase: PHASE1, PHASE2 • Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. • Aim: Disease control

Last updated May 26, 2026 12:12 UTC

GAUCHER DISEASE TYPE 3

Get alerted when new GAUCHER DISEASE TYPE 3 trials appear

Gene therapy injection aims to control rare brain disease in kids