Breakthrough gene therapy trial offers hope for rare genetic disorder
NCT ID NCT07135050
Summary
This early-stage study is testing a new gene therapy called MZ-1866 for people with Pitt Hopkins Syndrome, a rare genetic disorder that affects brain development. Twelve children and adults will receive a single injection of the therapy directly into the fluid around the brain. Researchers will monitor safety and look for any changes in development and symptoms over time.
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Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact
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Rush University Medical Center
NOT_YET_RECRUITINGChicago, Illinois, 60612, United States
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UCSF Benioff Children's Hospital Oakland
RECRUITINGOakland, California, 94609, United States
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Contact Email: •••••@•••••
Conditions
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