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.

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 ...

Health officials are trying to find ways to ensure equitable access to the breakthrough, high-cost gene therapies for sickle cell disease. The disease, which can cause bouts of debilitating pain, ...