Investigator Profile: Marie Scully, M.D, Professor of Hematology at University College London Hospitals

Mar 22, 2019 | Investigator Content, Scully, Marie M.D Content

Investigator Profile Immunology Researcher

Dr. Scully was the lead author of the HERCULES study. The objective of the Phase 3 study pre approval was to evaluate the long term-safety and efficacy of repeated use of caplacizumab (Cablivi® ) and to characterize long term impact of TTP. TTP is a rare, life threatening autoimmune blood disorder characterized by extensive clot formation in small blood vessels throughout the body leading to severe thrombocytopenia (very low platelet count), microangiopathic hemolytic anemia, (loss of red blood cells through destruction), ischemia (restricted blood supply to parts of the body) and widespread organ damage, especially in the brain and heart. Key findings are included here. The FDA approved the drug February 2019. The sponsor Ablynx was acquired by Sanofi in late 2018.

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