Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Conditions
Beta-Thalassemia · Thalassemia · Hematologic Diseases · Genetic Diseases, Inborn · Hemoglobinopathies · Sickle Cell Disease · Sickle Cell Anemia
Simple summaryAI-assisted
This summary is produced by a language model and is experimental. It may be incomplete or inaccurate and is not a substitute for professional medical advice.
Locations
New York Presbyterian Hospital - Morgan Stanley Children's Hospital, New York, New York, United States
Levine Children's Hospital - Hematology, Charlotte, North Carolina, United States
TriStar Medical Group Children's Specialists - Pediatric Oncology, Nashville, Tennessee, United States
University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology, Düsseldorf, Germany
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica, Rome, Italy
King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology, Al Mathar Ash Shamali, Saudi Arabia