Tracking the future: 5-Year watch for kids in groundbreaking brain disease gene therapy trial
NCT ID NCT06614569
Summary
This study is monitoring 7 children 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 years after the treatment. Researchers will also track how the children are doing to see if the therapy changes the expected course of these severe diseases.
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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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UMass Chan Medical School
Worcester, Massachusetts, 01655, United States
Conditions
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