Associated Papers
Advances in Drug Discovery Targeting Lysosomal Membrane Proteins.
Wang H et al.
Pharmaceuticals (basel, Switzerland), 2023
In Vitro and In Vivo Models to Study Nephropathic Cystinosis.
Cheung PY et al.
Cells, 2021
ELX-02: an investigational read-through agent for the treatment of nonsense mutation-related genetic disease.
Kerem E
Expert Opinion on Investigational Drugs, 2020
The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis.
Brasell EJ et al.
Plos One, 2019
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