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.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Study contacts
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Contact
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