Cystinosis is a prototypical lysosomal storage disease caused by recessive pathogenic variants in CTNS, which encodes cystinosin, a ubiquitous proton-driven lysosomal cystine transporter.1 Loss of cystinosin leads to intralysosomal cystine accumulation, impaired lysosomal signaling, defective autophagy, and widespread cellular dysfunction.2-4 The most severe and…
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