Gene therapy trial hopes to ease angelman syndrome symptoms
NCT ID NCT07181837
First seen Nov 01, 2025 · Last updated May 25, 2026 · Updated 31 times
Summary
This study tests a new gene therapy called MVX-220 for people with Angelman syndrome, a genetic condition that causes severe developmental delays. The therapy is given as a single injection into the fluid around the brain. The trial will include 12 children and adults to see if it is safe and if it can improve communication and other symptoms.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cedars-Sinai Medical Center
RECRUITINGLos Angeles, California, 90048, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Rush University Medical Center
RECRUITINGChicago, Illinois, 60612, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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