Scientists test gene therapy for devastating childhood brain disease
NCT ID NCT04669535
Summary
This early-stage trial tested a gene therapy called AXO-AAV-GM2 in children with Tay-Sachs or Sandhoff disease, rare and fatal genetic disorders that destroy the brain. The study aimed to see if delivering working copies of the faulty genes directly into the brain and spinal fluid was safe and could slow the disease. It enrolled 9 children, ranging from infants to young teenagers, but was terminated before completion.
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Contacts and locations
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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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