New hope for rare blood disorder: study reviews real-world use of enzyme therapy
NCT ID NCT07429942
First seen Jun 27, 2026 · Last updated Aug 07, 2026 · Updated 2 times
Summary
This study looks back at medical records of people with congenital thrombotic thrombocytopenic purpura (cTTP), a rare inherited blood disorder that causes dangerous blood clots. Participants received a manufactured version of the missing ADAMTS13 enzyme through an early access program. Researchers will compare episodes and symptoms before and during treatment to see how well the therapy works in real life.
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Conditions
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