The first participant has now been dosed in a global clinical trial designed to confirm the benefits of mitapivat,…
Articles by Michela Luciano, PhD
Agios Pharmaceuticals is discontinuing development of its oral therapy candidate, tebapivat, for people with sickle cell disease (SCD). The…
Casgevy (exagamglogene autotemcel) is now approved in the U.S. to treat children as young as 2 with sickle cell…
Fulcrum Therapeutics said it is discontinuing development of pociredir, its experimental oral treatment for sickle cell disease (SCD),…
An experimental, single-dose gene-editing therapy from Tessera Therapeutics successfully corrected the genetic mutation that causes sickle cell disease…
Children with sickle cell anemia (SCA), the most common and severe form of sickle cell disease (SCD), show signs…
The Food and Drug Administration (FDA) has granted breakthrough device designation to the Gazelle…
The breakdown of red blood cells — a defining feature of sickle cell disease (SCD) — may contribute to…
Big Nova has made a $3 million donation to support NMDP’s efforts to help more patients, including those with…
An intervention designed to improve adherence to hydroxyurea — by providing extra support from community health workers (CHWs) and…