Scientists track patients for 15 years after rare disease gene therapy
NCT ID NCT05506254
Summary
This study is monitoring the long-term safety of four patients who previously received an experimental gene therapy (hLB-001) for a rare genetic disorder called methylmalonic acidemia (MMA). The main goal is to check for any side effects that may appear over a very long period—up to 15 years. This is a follow-up study only; patients are not receiving any new treatment here.
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Contacts and locations
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Locations
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Clinical Trial Site
Atlanta, Georgia, 30322, United States
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Clinical Trial Site
Pittsburgh, Pennsylvania, 15224, United States
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Clinical Trial Site
Nashville, Tennessee, 37232, United States
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Clinical Trial Site
Seattle, Washington, 98105, United States
Conditions
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