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Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression.
Proceedings of the National Academy of Sciences of the United States of America 20161101
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Proposing a mechanism of action for ataluren.
Proceedings of the National Academy of Sciences of the United States of America 20161101
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Orphan drug development in muscular dystrophy: update on two large clinical trials of dystrophin rescue therapies.
Discovery medicine 20131101
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A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays.
PLoS biology 20130601
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A comparative evaluation of NB30, NB54 and PTC124 in translational read-through efficacy for treatment of an USH1C nonsense mutation.
EMBO molecular medicine 20121101
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Gene therapy for muscular dystrophy: lessons learned and path forward.
Neuroscience letters 20121011
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Cystic fibrosis: insight into CFTR pathophysiology and pharmacotherapy.
Clinical biochemistry 20121001
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Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy.
Human molecular genetics 20120915
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Highlights of the North American Cystic Fibrosis Conference 2011.
Journal of the Royal Society of Medicine 20120601
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Rescue of melanocortin 4 receptor (MC4R) nonsense mutations by aminoglycoside-mediated read-through.
Obesity (Silver Spring, Md.) 20120501
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Drug treatment of Duchenne muscular dystrophy: available evidence and perspectives.
Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology 20120501
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The chemical compound PTC124 does not affect cellular electrophysiology of cardiac ventricular myocytes.
Cardiovascular drugs and therapy 20120201
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Personalized medicine comes to cystic fibrosis.
American journal of medical genetics. Part A 20120201
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Cystic fibrosis transmembrane conductance regulator-modifying medications: the future of cystic fibrosis treatment.
The Annals of pharmacotherapy 20120101
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Stop codon read-through with PTC124 induces palmitoyl-protein thioesterase-1 activity, reduces thioester load and suppresses apoptosis in cultured cells from INCL patients.
Molecular genetics and metabolism 20111101
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[Therapeutic readthrough strategy for suppression of nonsense mutations in duchenne muscular dystrophy].
Brain and nerve = Shinkei kenkyu no shinpo 20111101
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Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis.
The European respiratory journal 20110701
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PTC124-mediated translational readthrough of a nonsense mutation causing Usher syndrome type 1C.
Human gene therapy 20110501
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Nonsense suppressor therapies rescue peroxisome lipid metabolism and assembly in cells from patients with specific PEX gene mutations.
Journal of cellular biochemistry 20110501
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Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
Molecular therapy : the journal of the American Society of Gene Therapy 20110501
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PTC124 improves readthrough and increases enzymatic activity of the CPT1A R160X nonsense mutation.
Journal of inherited metabolic disease 20110401
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Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse.
PloS one 20110101
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Nonsense-mediated mRNA decay and cystic fibrosis.
Methods in molecular biology (Clifton, N.J.) 20110101
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Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
American journal of respiratory and critical care medicine 20101115
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Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosis.
Current opinion in pulmonary medicine 20101101
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Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).
Journal of child neurology 20100901
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Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression.
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UGA hopping: a sport for nephrologists too?
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Molecule of the month. Ataluren.
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Protein biomarkers in cystic fibrosis research: where next?
Genome medicine 20100101
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Emerging genetic therapies to treat Duchenne muscular dystrophy.
Current opinion in neurology 20091001
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Emerging treatments in cystic fibrosis.
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Update in cystic fibrosis 2008.
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Drug developers aim to treat cystic fibrosis through disease modification.
American journal of health-system pharmacy : AJHP : official journal of the American Society of Health-System Pharmacists 20081215
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New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals.
Current opinion in allergy and clinical immunology 20081201
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Introducing sense into nonsense in treatments of human genetic diseases.
Trends in genetics : TIG 20081101
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Gunvalson decision sends shockwaves through industry.
Nature biotechnology 20081101
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Reading through premature stop codons with PTC1 24. Project Catalyst to find more Duchenne drugs. Interview by Guenter Scheuerbrandt.
Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology 20081001
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Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.
Lancet (London, England) 20080830
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Ignoring the nonsense: a phase II trial in cystic fibrosis.
Lancet (London, England) 20080830
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Pharmacological probing of type 1 autism.
Journal of autism and developmental disorders 20080801
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PTC124, nonsense mutations and Duchenne muscular dystrophy.
Neuromuscular disorders : NMD 20071001
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Studies point way to new therapeutic prospects for muscular dystrophy.
JAMA 20070926
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PTC124 targets genetic disorders caused by nonsense mutations.
Nature 20070503
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Chemical biology: ignore the nonsense.
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Emerging drug treatments for cystic fibrosis.
Expert opinion on emerging drugs 20070501
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Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.
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Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy.
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