Gene therapy trial hopes to improve communication in angelman syndrome
NCT ID NCT07181837
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 30 times
Summary
This study tests a new gene therapy called MVX-220 for people with Angelman syndrome, a genetic condition that affects the nervous system. The therapy is given as a single injection into the fluid around the brain and spinal cord. The trial will include 12 children (ages 4-8) and adults (ages 18-50) with specific genetic causes of Angelman syndrome. Researchers will check for side effects and measure changes in communication ability using a caregiver questionnaire.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for ANGELMAN SYNDROME are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Cedars-Sinai Medical Center
RECRUITINGLos Angeles, California, 90048, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Rush University Medical Center
RECRUITINGChicago, Illinois, 60612, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.