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- UniQure tumbles with latest data for Huntington’s gene therapyAnalysts guessed that investors wanted to see stronger durability results at a four-year check-in of the key trial supporting a potential approval of "AMT-130."BioPharma Dive · 21d ago↗ source
- FDA approves Emcitate for peripheral thyrotoxicosis in patients with MCT8 deficiencyThe U.S. Food and Drug Administration announced approval of Emcitate (tiratricol) tablets formulated for oral suspension to treat peripheral thyrotoxicosis in patients with MCT8 deficiency, also ca…