Brain injection gene therapy offers hope for rare neurological disorder
NCT ID NCT07181837
Summary
This early-stage trial is testing whether a single injection of experimental gene therapy (MVX-220) into the fluid around the brain can help people with Angelman syndrome. The study will enroll 12 children and adults to first check safety, then see if the treatment improves communication, development, and daily functioning. All participants will receive the gene therapy and be followed for five years to monitor long-term effects.
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Contacts and locations
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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
Explore the condition pages connected to this study.