Scientists test gene therapy for devastating childhood brain disease
NCT ID NCT04669535
Summary
This early-stage study tested the safety of an experimental gene therapy for children with Tay-Sachs or Sandhoff disease, rare and fatal genetic disorders that destroy the brain and nervous system. The therapy aimed to deliver working copies of the faulty genes directly into the brain and spinal fluid. The trial was terminated after enrolling 9 children, focusing first on finding a safe dose.
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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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Massachusetts General Hospital, Center for Rare Neurological Diseases
Boston, Massachusetts, 02114, United States
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University of Massachusetts Medical Health Center
Worcester, Massachusetts, 01655, United States
Conditions
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