识破谬论：庆大霉素和ELX - 02作为大疱性表皮松解症的挽救疗法 - Suppr

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超能文献

Reading through the nonsense: Gentamicin and ELX-02 as rescuing therapies for epidermolysis bullosa.

作者信息

Anderson-Crannage Morgan, Cairo Mitchell S, Liao Yanling

机构信息

Department of Pediatrics, New York Medical College, Valhalla, NY 10595, USA.

Department of Cell Biology and Anatomy, New York Medical College, Valhalla, NY 10595, USA.

出版信息

Mol Ther Nucleic Acids. 2024 Nov 13;35(4):102368. doi: 10.1016/j.omtn.2024.102368. eCollection 2024 Dec 10.

DOI:10.1016/j.omtn.2024.102368

PMID:39629116

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11613416/

Abstract

摘要

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7c9d/11613416/c35e873e3d2f/gr1.jpg

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本文引用的文献

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JAMA Dermatol. 2022 Apr 1;158(4):366-374. doi: 10.1001/jamadermatol.2021.5992.

A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Escalation Study to Evaluate the Safety and Pharmacokinetics of ELX-02 in Healthy Subjects.一项评估ELX-02在健康受试者中安全性和药代动力学的随机、双盲、安慰剂对照、多剂量递增研究。

Clin Pharmacol Drug Dev. 2021 Aug;10(8):859-869. doi: 10.1002/cpdd.914. Epub 2021 Jan 19.

Phase 1 Renal Impairment Trial Results Supports Targeted Individualized Dosing of ELX-02 in Patients With Nephropathic Cystinosis.1 期肾功能损害试验结果支持对胱氨酸病患者进行 ELX-02 的靶向个体化剂量给药。

J Clin Pharmacol. 2021 Jul;61(7):923-931. doi: 10.1002/jcph.1807. Epub 2021 Jan 18.

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Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers.ELX-02 单剂量递增给药的安全性、耐受性和药代动力学研究，ELX-02 是一种治疗由无义突变引起的遗传疾病的潜在药物，在健康志愿者中进行。

Clin Pharmacol Drug Dev. 2019 Nov;8(8):984-994. doi: 10.1002/cpdd.647. Epub 2019 Jan 16.

J Clin Invest. 2017 Aug 1;127(8):3028-3038. doi: 10.1172/JCI92707. Epub 2017 Jul 10.

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