Gene therapy patients monitored for years in rare disease study
NCT ID NCT03970278
Summary
This study followed 12 adults with a rare genetic liver disorder (GSDIa) for 4 to 6 years after they received a single dose of an experimental gene therapy. The main goal was to check for any long-term safety issues from the treatment. Researchers also monitored whether the therapy continued to help patients go longer without dangerously low blood sugar during fasting tests.
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Contacts and locations
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Locations
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Complejo Hospitalario Universitario de Santiago
Santiago de Compostela, A Coruna, 15706, Spain
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Michigan Medicine University of Michigan
Ann Arbor, Michigan, 48109, United States
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Montreal Children Hospital, McGill University Health Centre
Montreal, Quebec, H4A3J1, Canada
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UCONN Health
Farmington, Connecticut, 06030-3213, United States
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University Medical Center Groningen
Groningen, 9700RB, Netherlands
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University of Texas Health Science Center at Houston
Houston, Texas, 77030, United States
Conditions
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