Results 111 to 120 of about 1,505 (151)

Ataluren Treatment Improves Hematopoietic and Pancreatic Disorders in Patients with Shwachman-Diamond Syndrome

Cipolli M, Bezzerri V, Pegoraro A, Hristodor A, Crane G, Meneghelli I, Brignole C, Boni C, Baldisseri E, Vella A, Menichetti G, Valli R, Porta G, D'Amico G, Tecchio C, Parisi A, Lippi G, Cesaro S, Corey S.   +18 more
europepmc   +1 more source

Effect of Ataluren on dystrophin mutations [PDF]

Journal of Cellular and Molecular Medicine, 2020
AbstractDuchenne muscular dystrophy is a severe muscle wasting disease caused by mutations in the dystrophin gene (dmd). Ataluren has been approved by the European Medicines Agency for treatment of Duchenne muscular dystrophy. Ataluren has been reported to promote ribosomal read‐through of premature stop codons, leading to restoration of full‐length ...
Peter D. Currie, Mei Li, Joachim Berger
exaly   +3 more sources

Ataluren suppresses a premature termination codon in an MPS I-H mouse

Journal of Molecular Medicine, 2022
Abstract Suppressing translation termination at premature termination codons (PTCs), termed readthrough, is a potential therapy for genetic diseases caused by nonsense mutations. Ataluren is a compound that has shown promise for clinical use as a readthrough agent.
Marla Weetall, Kim M Keeling, Xiaojiao Xue   +2 more
exaly   +4 more sources

Ataluren as an Agent for Therapeutic Nonsense Suppression [PDF]

Annual Review of Medicine, 2013
The interplay of translation and mRNA turnover has helped unveil how the regulation of gene expression is a continuum in which events that occur during the birth of a transcript in the nucleus can have profound effects on subsequent steps in the cytoplasm.
Allan Jacobson, Stuart W Peltz, Ellen M Welch   +2 more
exaly   +3 more sources

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Synthesis and characterization of ataluren-cyclodextrins complexes

Journal of Molecular Structure, 2023
Seyed Saeied Homami, Amirhossein Monzavi
exaly   +2 more sources

Ataluren and aminoglycosides stimulate read-through of nonsense codons by orthogonal mechanisms [PDF]

Proceedings of the National Academy of Sciences of the United States of America, 2021
Significance Nonsense mutations giving rise to premature stop codons (PSCs) cause many diseases, creating the need to develop safe and effective translational read-through–inducing drugs (TRIDs). The current best-characterized TRIDs are ataluren and aminoglycosides. Only ataluren has been approved for clinical use, albeit in a limited
Martin Y Ng, Barry S Cooperman, Yale E. Goldman   +2 more
exaly   +3 more sources

LC–MS/MS Quantification of Ataluren and Ataluren Acyl Glucuronide in Human Plasma/Urine: Application in Clinical Studies

Bioanalysis, 2020
Background: This paper describes for the first-time analytical procedures established to resolve the challenges associated with simultaneous and direct quantification of ataluren and ataluren-O-1β-acyl glucuronide (AAG) by LC-MS/MS in human plasma and urine matrices.
Guodong Gu, Ronald Kong, Jiyuan Ma
exaly   +3 more sources