New drug offers hope for brain symptoms in rare gaucher disease
NCT ID NCT07675031
First seen Jun 30, 2026 · Last updated Jul 01, 2026 · Updated 1 time
Summary
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 goal is to offer a potential treatment for the neurological aspects of the disease, which currently have no approved therapies.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- venglustat
- What this could lead to
- If venglustat works, it could offer a treatment option for the brain-related symptoms of Type 3 Gaucher disease, for which no approved therapies currently exist.
- What could go wrong
- This is an early-access program, not a formal trial, so data on effectiveness and safety are still limited. The drug may not work for all patients or could have side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for TYPE 3 GAUCHER DISEASE are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Email: •••••@•••••