Exagamglogene Autotemcel for Severe Sickle Cell Disease.

Haydar Frangoul Franco Locatelli Akshay SharmaMonica BhatiaMarkus MaparaLyndsay MolinariDonna WallRobert I LiemPaul TelferAmi J ShahMarina CavazzanaSelim CorbaciogluDamiano RondelliRoland MeiselLaurence DedekenStephan LobitzMariane de MontalembertMartin H SteinbergMark C WaltersMichael J EckrichSuzan ImrenLaura BowerChristopher SimardWeiyu ZhouFengjuan XuanPhuong Khanh MorrowWilliam E HobbsStephan A Gruppnull null

Published in: The New England journal of medicine (2024)

Treatment with exa-cel eliminated vaso-occlusive crises in 97% of patients with sickle cell disease for a period of 12 months or more. (CLIMB SCD-121; ClinicalTrials.gov number, NCT03745287.).

Keyphrases

sickle cell disease
end stage renal disease
newly diagnosed
ejection fraction
peritoneal dialysis
prognostic factors
early onset
patient reported outcomes
combination therapy
drug induced