Gene therapy brings light to rare eye disease in japanese patients
NCT ID NCT04516369
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a gene therapy called voretigene neparvovec in 4 Japanese patients with a rare inherited eye disease that causes vision loss. The treatment was given as an injection under the retina to replace the faulty gene. The goal was to see if it could safely improve light sensitivity and visual field.
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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.
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Locations
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Novartis Investigative Site
Meguro-ku, Tokyo, 152-8902, Japan