Results 121 to 130 of about 1,505 (151)

Ataluren Pharmacokinetics in Healthy Japanese and Caucasian Subjects [PDF]

Clinical Pharmacology in Drug Development, 2019
AbstractTo evaluate the potential for ethnicity‐related differences in ataluren pharmacokinetics (PK) and safety, a phase 1 single‐dose study was conducted in 48 healthy (24 Japanese and 24 Caucasian subjects), nonsmoking male volunteers who were equally divided into 3 cohorts of oral doses at 5, 10, and 20 mg/kg.
Ronald Kong, Joseph McIntosh
exaly   +3 more sources

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Ataluren for the treatment of cystic fibrosis

Expert Review of Respiratory Medicine, 2016
Alleles causing diseases that carry premature termination codons (PTCs) will cause premature cessation of translation, leading to loss of function and consequent disease. Recently, a novel agent, Ataluren, was developed through a high throughput screening program.
David, Shoseyov, Malena, Cohen-Cymberknoh, Michael, Wilschanski   +2 more
openaire   +2 more sources

Ataluren for the Treatment of Usher Syndrome 2A Caused by Nonsense Mutations [PDF]

International Journal of Molecular Sciences, 2019
The identification of genetic defects that underlie inherited retinal diseases (IRDs) paves the way for the development of therapeutic strategies. Nonsense mutations caused approximately 12% of all IRD cases, resulting in a premature termination codon (PTC).
Kerstin Nagel-Wolfrum, Susanne Kohl, Katarina Stingl   +2 more
exaly   +3 more sources

Ataluren: First Global Approval

Drugs, 2014
Nonsense mutations are implicated in 5-70 % of individual cases of most inherited diseases, including Duchenne muscular dystrophy (DMD) and cystic fibrosis. Ataluren (Translarna™) is an orally available, small molecule compound that targets nonsense mutations, and is the first drug in its class.
openaire   +2 more sources

Enhancement of premature stop codon readthrough in the CFTR gene by Ataluren (PTC124) derivatives

European Journal of Medicinal Chemistry, 2015
Premature stop codons are the result of nonsense mutations occurring within the coding sequence of a gene. These mutations lead to the synthesis of a truncated protein and are responsible for several genetic diseases. A potential pharmacological approach to treat these diseases is to promote the translational readthrough of premature stop codons by ...
Ivana Pibiri, Laura Lentini, Raffaella Melfi, Giulia Gallucci, Andrea Pace, Angelo Spinello, Giampaolo Barone, Aldo Di Leonardo   +7 more
openaire   +2 more sources

Ataluren

Drugs of the Future, 2008
null Davies, S., null Serradell, N., null Rosa, E., null Castaner, R.   +3 more
openaire   +1 more source

Ataluren metabolism: Ataluren--1β-acyl glucuronide is a stable circulating metabolite in mouse, rat, dog and human

Drug Metabolism and Pharmacokinetics, 2021
Joseph M Colacino, Marla Weetall, Ronald Kong   +2 more
exaly  

A combined molecular docking and molecular dynamics simulation approach to probing the host–guest interactions of Ataluren with natural and modified cyclodextrins

Molecular Simulation, 2022
Seyed Saeied Homami, Farhad Bayat, Amirhossein Monzavi   +2 more
exaly  

Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)

Journal of Cystic Fibrosis, 2020
Michael W Konstan, Eitan Kerem, Donald R. VanDevanter   +2 more
exaly  

Ataluren prevented bone loss induced by ovariectomy and aging in mice through the BMP-SMAD signaling pathway

Biomedicine and Pharmacotherapy, 2023
Zhengwei Xie, Yongsheng Zhou, Ranli Gu
exaly