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- UniQure crashes as Huntington’s gene therapy shows less slowing of disease at 4 yearsThe results are nevertheless “unprecedented,” UniQure executives said on an investor call Tuesday, emphasizing that the updated natural history database used as the control group for the Phase 1/2…BioSpace · 22d 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…