15-Year watch begins on pioneering gene therapy for blood disorder
NCT ID NCT06717932
Summary
This study is tracking the long-term safety and effectiveness of a gene therapy called CS-101 for people with beta-thalassemia, a serious inherited blood disorder. It will follow 8 participants who received the therapy in a previous trial for up to 15 years. The main goals are to monitor for any long-term side effects and see if the treatment continues to work, including whether patients remain free from needing blood transfusions.
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Contacts and locations
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Locations
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The First Affiliated Hospital of Guangxi Medical University
Nanning, China
Conditions
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