ELX-02

01/01/2021 - " results of this study support the evaluation of ELX-02 in phase 2 clinical trials with patients that have genetic diseases caused by nonsense mutations."
01/01/2020 - "Thus, ELX-02 may be a potential therapeutic option for nonsense mutation-mediated genetic diseases."
11/01/2019 - "Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers."
11/01/2019 - "ELX-02 is being developed as a therapy for genetic diseases caused by nonsense mutations. "
01/01/2021 - "ELX-02 is an investigational compound being developed as a therapy for genetic diseases caused by nonsense mutations such as cystic fibrosis. "

01/01/2021 - "An estimated glomerular filtration rate (eGFR)-PK model of ELX-02 was developed from the RI study data and used to implement individualized dosing in a phase 2a study in patients with nephropathic cystinosis to achieve a weekly targeted exposure (area under the plasma concentration-time curve [AUC]). "
01/01/2021 - "The aim of this study was to assess the pharmacokinetics (PK) and safety of ELX-02 in a renally impaired population and apply these findings to the individualized dosing of patients with nephropathic cystinosis. "
01/01/2021 - "Phase 1 Renal Impairment Trial Results Supports Targeted Individualized Dosing of ELX-02 in Patients With Nephropathic Cystinosis."
01/01/2021 - "ELX-02 was well tolerated by RI and nephropathic cystinosis populations. "
01/01/2019 - "The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis."

01/01/2021 - "ELX-02 is an investigational compound being developed as a therapy for genetic diseases caused by nonsense mutations such as cystic fibrosis. "

11/01/2019 - "Single subcutaneously injected doses of ELX-02 between 0.3 mg/kg and 7.5 mg/kg showed an acceptable safety profile without severe or serious drug-related adverse events, including a lack of renal and ototoxicity events. "