Phase 1 Renal Impairment Trial Results Supports Targeted Individualized Dosing of ELX‐02 in Patients With Nephropathic Cystinosis
Author:
Affiliation:
1. Eloxx Pharmaceuticals Waltham Massachusetts USA
2. Syneos Health Miami Florida USA
3. Pharmacokinetics Consultant Eloxx Pharmaceuticals Waltham Massachusetts USA
4. McGill University Montreal Quebec Canada
Publisher
Wiley
Subject
Pharmacology (medical),Pharmacology
Link
https://onlinelibrary.wiley.com/doi/pdf/10.1002/jcph.1807
Reference13 articles.
1. Increased Selectivity toward Cytoplasmic versus Mitochondrial Ribosome Confers Improved Efficiency of Synthetic Aminoglycosides in Fixing Damaged Genes: A Strategy for Treatment of Genetic Diseases Caused by Nonsense Mutations
2. When proteins start to make sense: fine-tuning of aminoglycosides for PTC suppression therapy
3. The aminoglycoside geneticin permits translational readthrough of the CTNS W138X nonsense mutation in fibroblasts from patients with nephropathic cystinosis
4. Synthetic Aminoglycosides Efficiently Suppress Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations and Are Enhanced by Ivacaftor
5. The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis
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