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The FDA has granted orphan drug designation to the next generation BCMA-directed CAR T-cell therapy NXC-201 for the treatment of patients with amyloid light chain amyloidosis. ....
Dr Kremyanskaya discusses the results from cohort 1 of the MANIFEST trial in treatment-naïve myelofibrosis, differentiating features of pelabresib and recently approved JAK inhibitors, and next steps for the investigation of BET inhibitors in this population. ....