[1]RussianChemicalBulletin,2015,vol.64,#1,p.142-145
[2]Izv.Akad.Nauk,Ser.Khim.,2015,#1,p.142-145,4
[3]NewJournalofChemistry,2014,vol.38,#7,p.3062-3070
[1]Patent:US2004/204461,2004,A1,.Locationinpatent:Page54
[2]Patent:WO2007/117438,2007,A2,.Locationinpatent:Page/Pagecolumn261
[3]Patent:WO2008/45566,2008,A1,.Locationinpatent:Page/Pagecolumn25-26
[4]Patent:WO2006/110483,2006,A1,.Locationinpatent:Page/Pagecolumn21
[5]AdvancedSynthesisandCatalysis,2017,vol.359,#5,p.772-778
[6]Patent:WO2008/30570,2008,A1,.Locationinpatent:Page/Pagecolumn20-21
[7]Patent:WO2008/30570,2008,A1,.Locationinpatent:Page/Pagecolumn21-23
[8]BioorganicandMedicinalChemistryLetters,2014,vol.24,#11,p.2473-2476
[9]Patent:CN105461650,2016,A,.Locationinpatent:Paragraph0075;0082
[1]Patent:CN106279057,2017,A,.Locationinpatent:Paragraph0023;0024
[1]Patent:WO2008/30570,2008,A1,.Locationinpatent:Page/Pagecolumn23-24
[1]Patent:CN105461650,2016,A,
[2]AdvancedSynthesisandCatalysis,2017,vol.359,#5,p.772-778
[3]Patent:WO2006/110483,2006,A1,
[1]Patent:WO2008/30570,2008,A1.Locationinpatent:Page/Pagecolumn23-24
[1]Patent:US2004/204461,2004,A1.Locationinpatent:Page54
[2]Patent:WO2015/134711,2015,A1.Locationinpatent:Paragraph00245
[1]Patent:US2004/204461,2004,A1.Locationinpatent:Page54
[2]Patent:WO2007/117438,2007,A2.Locationinpatent:Page/Pagecolumn261
[3]Patent:WO2008/45566,2008,A1.Locationinpatent:Page/Pagecolumn25-26
[4]Patent:WO2006/110483,2006,A1.Locationinpatent:Page/Pagecolumn21
[5]AdvancedSynthesisandCatalysis,2017,vol.359,p.772-778
[6]Patent:WO2008/30570,2008,A1.Locationinpatent:Page/Pagecolumn20-21
[7]Patent:WO2008/30570,2008,A1.Locationinpatent:Page/Pagecolumn21-23
[8]BioorganicandMedicinalChemistryLetters,2014,vol.24,p.2473-2476
[9]Patent:CN105461650,2016,A.Locationinpatent:Paragraph0075;0082
[1]ProceedingsoftheNationalAcademyofSciencesoftheUnitedStatesofAmerica,2010,vol.107,p.4878-4883
[1]BioorganicandMedicinalChemistryLetters,2014,vol.24,p.2473-2476
Title: Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression.
Journal: Proceedings of the National Academy of Sciences of the United States of America 20161101
Title: Proposing a mechanism of action for ataluren.
Journal: Proceedings of the National Academy of Sciences of the United States of America 20161101
Title: Orphan drug development in muscular dystrophy: update on two large clinical trials of dystrophin rescue therapies.
Journal: Discovery medicine 20131101
Title: A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays.
Journal: PLoS biology 20130601
Title: A comparative evaluation of NB30, NB54 and PTC124 in translational read-through efficacy for treatment of an USH1C nonsense mutation.
Journal: EMBO molecular medicine 20121101
Title: Gene therapy for muscular dystrophy: lessons learned and path forward.
Journal: Neuroscience letters 20121011
Title: Cystic fibrosis: insight into CFTR pathophysiology and pharmacotherapy.
Journal: Clinical biochemistry 20121001
Title: Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy.
Journal: Human molecular genetics 20120915
Title: Highlights of the North American Cystic Fibrosis Conference 2011.
Journal: Journal of the Royal Society of Medicine 20120601
Title: Rescue of melanocortin 4 receptor (MC4R) nonsense mutations by aminoglycoside-mediated read-through.
Journal: Obesity (Silver Spring, Md.) 20120501
Title: Drug treatment of Duchenne muscular dystrophy: available evidence and perspectives.
Journal: Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology 20120501
Title: The chemical compound PTC124 does not affect cellular electrophysiology of cardiac ventricular myocytes.
Journal: Cardiovascular drugs and therapy 20120201
Title: Personalized medicine comes to cystic fibrosis.
Journal: American journal of medical genetics. Part A 20120201
Title: Cystic fibrosis transmembrane conductance regulator-modifying medications: the future of cystic fibrosis treatment.
Journal: The Annals of pharmacotherapy 20120101
Title: Stop codon read-through with PTC124 induces palmitoyl-protein thioesterase-1 activity, reduces thioester load and suppresses apoptosis in cultured cells from INCL patients.
Journal: Molecular genetics and metabolism 20111101
Title: [Therapeutic readthrough strategy for suppression of nonsense mutations in duchenne muscular dystrophy].
Journal: Brain and nerve = Shinkei kenkyu no shinpo 20111101
Title: Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis.
Journal: The European respiratory journal 20110701
Title: PTC124-mediated translational readthrough of a nonsense mutation causing Usher syndrome type 1C.
Journal: Human gene therapy 20110501
Title: Nonsense suppressor therapies rescue peroxisome lipid metabolism and assembly in cells from patients with specific PEX gene mutations.
Journal: Journal of cellular biochemistry 20110501
Title: Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy 20110501
Title: PTC124 improves readthrough and increases enzymatic activity of the CPT1A R160X nonsense mutation.
Journal: Journal of inherited metabolic disease 20110401
Title: Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse.
Journal: PloS one 20110101
Title: Nonsense-mediated mRNA decay and cystic fibrosis.
Journal: Methods in molecular biology (Clifton, N.J.) 20110101
Title: Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
Journal: American journal of respiratory and critical care medicine 20101115
Title: Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosis.
Journal: Current opinion in pulmonary medicine 20101101
Title: Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).
Journal: Journal of child neurology 20100901
Title: Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression.
Journal: Journal of applied physiology (Bethesda, Md. : 1985) 20100901
Title: [Research on mRNA degradation and drug discovery].
Journal: Nihon yakurigaku zasshi. Folia pharmacologica Japonica 20100901
Title: UGA hopping: a sport for nephrologists too?
Journal: Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association 20100801
Title: Molecule of the month. Ataluren.
Journal: Drug news & perspectives 20100301
Title: Protein biomarkers in cystic fibrosis research: where next?
Journal: Genome medicine 20100101
Title: Emerging genetic therapies to treat Duchenne muscular dystrophy.
Journal: Current opinion in neurology 20091001
Title: Emerging treatments in cystic fibrosis.
Journal: Drugs 20091001
Title: [Mutation-specific treatments for Duchenne muscular dystrophy].
Journal: Brain and nerve = Shinkei kenkyu no shinpo 20090801
Title: PTC124 for cystic fibrosis.
Journal: Lancet (London, England) 20090425
Title: Update in cystic fibrosis 2008.
Journal: American journal of respiratory and critical care medicine 20090315
Title: Pharmaceuticals targeting nonsense mutations in genetic diseases: progress in development.
Journal: BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy 20090101
Title: Drug developers aim to treat cystic fibrosis through disease modification.
Journal: American journal of health-system pharmacy : AJHP : official journal of the American Society of Health-System Pharmacists 20081215
Title: New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals.
Journal: Current opinion in allergy and clinical immunology 20081201
Title: Introducing sense into nonsense in treatments of human genetic diseases.
Journal: Trends in genetics : TIG 20081101
Title: Gunvalson decision sends shockwaves through industry.
Journal: Nature biotechnology 20081101
Title: Reading through premature stop codons with PTC1 24. Project Catalyst to find more Duchenne drugs. Interview by Guenter Scheuerbrandt.
Journal: Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology 20081001
Title: Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.
Journal: Lancet (London, England) 20080830
Title: Ignoring the nonsense: a phase II trial in cystic fibrosis.
Journal: Lancet (London, England) 20080830
Title: Pharmacological probing of type 1 autism.
Journal: Journal of autism and developmental disorders 20080801
Title: PTC124, nonsense mutations and Duchenne muscular dystrophy.
Journal: Neuromuscular disorders : NMD 20071001
Title: Studies point way to new therapeutic prospects for muscular dystrophy.
Journal: JAMA 20070926
Title: PTC124 targets genetic disorders caused by nonsense mutations.
Journal: Nature 20070503
Title: Chemical biology: ignore the nonsense.
Journal: Nature 20070503
Title: Emerging drug treatments for cystic fibrosis.
Journal: Expert opinion on emerging drugs 20070501
Title: Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.
Journal: Journal of clinical pharmacology 20070401
Title: Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy.
Journal: IDrugs : the investigational drugs journal 20061101
Title: Nonsense mutations: running the red light.
Journal: Nature 20051208
Title: Pettit RS, et al. CFTR Modulators for the Treatment of Cystic Fibrosis. P T. 2014 Jul;39(7):500-11.
Title: Welch EM, et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature, 2007, 447(7140), 87-91.
Title: Miller JN, et al. The novel Cln1(R151X) mouse model of infantile neuronal ceroid lipofuscinosis (INCL) for testing nonsense suppression therapy. Hum Mol Genet. 2015 Jan 1;24(1):185-96.