FDA Approves Adzynma for Rare Blood Clotting Disorder cTTP
November 27th 2023CTTP is rare hereditary blood clotting disorder stemming from a disease-causing mutation in the ADAMTS13 gene. This gene plays a crucial role in producing the ADAMTS13 enzyme, responsible for regulating blood clotting.
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FDA Updates for the Week of Nov. 20, 2023
November 25th 2023FDA has approved a new therapy for HR positive breast cancer and announces plan to hold advisory meeting for Abecma in earlier treatment of multiple myeloma. In addition, Janssen has submitted a supplemental application for Rybrevant in NSCLC.
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Ten patients have been treated with the CRISPR-based gene therapy, the drug developer's chief scientific officer announced today, and high doses have had the intended effect of lowering LDL cholesterol. One study volunteer died five weeks after the infusion from cardiac arrest, but the company says the death was unrelated to the therapy and no changes were made to the study.
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