New gene therapy aims to tackle fabry disease in early trial
NCT ID NCT06539624
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy called EXG110 in 12 people with Fabry disease. The therapy uses a harmless virus to deliver a working gene that may help the body produce a missing enzyme. The main goal is to check safety and find the right dose, while also looking at how it affects kidney, heart, and skin symptoms.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
EXG110 (a gene therapy using a modified virus to deliver a working gene)
What this could lead to
If successful, this could point toward a one-time treatment that helps control Fabry disease symptoms and organ damage.
What could go wrong
This is a very early, small trial with only 12 people, so results may not apply widely. Gene therapies can have side effects like immune reactions or liver issues.
Disclaimer
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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.
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
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital, Zhejiang University School of Medicine
RECRUITINGHangzhou, Zhejiang, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Shanghai Children's Medical Center
RECRUITINGShanghai, Shanghai Municipality, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact