Gene therapy for cystinosis, a lysosomal storage disease, led to decreased white-cell cystine levels and stopping or decreasing cysteamine; adverse events were mainly consistent with the myeloablative regimen and underlying disease.
Hematology/Oncology
|11th Mar, 2026
|The New England Journal of Medicine
Hematology/Oncology
|11th Mar, 2026
|The New England Journal of Medicine
Hematology/Oncology
|11th Mar, 2026
|The New England Journal of Medicine
Hematology/Oncology
|11th Mar, 2026
|The New England Journal of Medicine
Hematology/Oncology
|11th Mar, 2026
|The New England Journal of Medicine
Hematology/Oncology
|11th Mar, 2026
|The New England Journal of Medicine
Hematology/Oncology
|11th Mar, 2026
|The New England Journal of Medicine