Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel.

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Title: Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel.
Authors: Sharma A; Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN., Locatelli F; IRCCS, Ospedale Pediatrico Bambino Gesù Rome, Catholic University of the Sacred Heart, Rome, Italy., Bhatia M; Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian Morgan Stanley Children's Hospital, New York, NY., Molinari L; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX., Mapara MY; Division of Hematology/Oncology, Department of Medicine, Columbia University, New York, NY., Liem RI; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL., Dedeken L; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels, Belgium., Wall D; The Hospital for Sick Children, University of Toronto, Toronto, Canada., Eckrich MJ; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX., Kuo KHM; University Health Network, Toronto, Canada., Smith W; Virginia Commonwealth University, Richmond, VA., Imren S; Vertex Pharmaceuticals Incorporated, Boston, MA., Kohli P; Vertex Pharmaceuticals Incorporated, Boston, MA., Li N; Vertex Pharmaceuticals Incorporated, Boston, MA., Liu T; Vertex Pharmaceuticals Incorporated, Boston, MA., Rubin J; Vertex Pharmaceuticals Incorporated, Boston, MA., Hobbs W; Vertex Pharmaceuticals Incorporated, Boston, MA., Grupp SA; Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA., Frangoul H; Sarah Cannon Research Institute at TriStar Centennial Children's Hospital, Nashville, TN.
Source: Blood advances [Blood Adv] 2025 Dec 23; Vol. 9 (24), pp. 6481-6490.
Publication Type: Clinical Trial, Phase III; Journal Article; Multicenter Study
Journal Info: Publisher: American Society of Hematology Country of Publication: United States NLM ID: 101698425 Publication Model: Print Cited Medium: Internet ISSN: 2473-9537 (Electronic) Linking ISSN: 24739529 NLM ISO Abbreviation: Blood Adv Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2473-9537
DOI:10.1182/bloodadvances.2025016701