Agios collapsed ~50% in a single day after its pivotal RISE UP sickle cell disease, SCD trial delivered a split verdict: it met the hemoglobin (Hb) response endpoint but failed to show a statistically ...
Some types of sickle cell disease cause severe illness, while others have a mild or unapparent course. Sickle cell disease is present from birth and inherited from genes you receive from your parents.
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