"Anemia eyes," or "anemic retinopathy," is a term that describes the vision changes caused by damage from anemia (a lack of ...

In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants ...

BEAM incurs a narrower-than-expected loss in the fourth quarter. The company remains focused on pipeline development.

In addition, improvements in changes from baseline for markers of hemolysis were observed in the mitapivat arm compared to ...