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Clinical classification, screening and diagnosis for thalassemia

10.1371/journal.pone.0120426 101 MicheletR.UrsinoM.BouletS.FranckS.CasilagF.BaldryM.et al (2021)

no large modern RCT exists

The patients who come to Perfect B for this protocol are not always high-level athletes

8a and Supplementary Figs

(Given the potential long-term consequences of NMOSD relapses, the company could not ethically include a direct placebo arm in its Phase III trial.) Ultomiris was well-tolerated overall, and most treatment-emergent events were mild or moderate in severity, according to AstraZeneca