Exagamglogene autotemcel (exa-cel), a CRISPR-Cas9 gene-edited cell therapy, led to sustained improvements in health-related quality of life (HRQOL) for both adults and adolescents with ...
(WASHINGTON — August 27, 2025) – Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or ...
Patients with sickle cell disease and beta-thalassemia treated with exagamglogene autotemcel reported substantial and ...
The new Prescription Drug User Fee Act target date is now December 7, 2025. The Food and Drug Administration (FDA) has extended the review period for the supplemental New Drug Application (sNDA) for ...
Thanks to groundbreaking in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births. However, this medical advancement created a new challenge: a ...
Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with the Italian Medicines Agency (AIFA) for eligible transfusion-dependent beta thalassemia (TDT) and severe sickle ...
In the beta thalassemia study, Dr. de la Fuente and his colleagues used two quality of life scales to evaluate outcomes in adult patients and two quality of life scales for adolescent patients. Unlike ...
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