Scientists track 7 patients for 5 years after groundbreaking gene therapy
NCT ID NCT06614569
Summary
This study monitors 7 patients for five years after they received an experimental gene therapy for Tay-Sachs or Sandhoff disease. The goal is to check for any long-term safety issues, like cancer or immune system problems, that might develop after treatment. Researchers will also track how the treatment affects the patients' brain function and development over time.
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Contacts and locations
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Locations
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UMass Chan Medical School
Worcester, Massachusetts, 01655, United States
Conditions
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