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To test if the stabilized lysosome form of cystinosin(7) was functional, we measured lysosomal cystine levels in patient fibroblasts after treating them with the correctors

In this protein adduct pathway NAPQI primarily targets mitochondrial proteins and ion channels, leading to the loss of energy production, disturbances in ionic balance, and ultimately cell death

Pfizer was hoping to throw its hat into the ring, but now it seems the pharma company has taken itself out of the running at least for now