Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.
about
Moving towards effective therapeutic strategies for Neuronal Ceroid LipofuscinosisTranslational readthrough potential of natural termination codons in eucaryotes--The impact of RNA sequenceMechanisms of granulin deficiency: lessons from cellular and animal modelsTherapeutic suppression of premature termination codons: mechanisms and clinical considerations (review)Animal models of hemophiliaCurrent understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophyThe ubiquitin-proteasome system and nonsense-mediated mRNA decay in hypertrophic cardiomyopathyIdentification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screeningTranslational readthrough by the aminoglycoside geneticin (G418) modulates SMN stability in vitro and improves motor function in SMA mice in vivoDiscovery of a Selective S1P1 Receptor Agonist Efficacious at Low Oral Dose and Devoid of Effects on Heart Rate.Dystrophic Cardiomyopathy-Potential Role of Calcium in Pathogenesis, Treatment and Novel TherapiesThe unfolded protein response affects readthrough of premature termination codonsGenetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy.Cystic fibrosis: exploiting its genetic basis in the hunt for new therapiesTherapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.Aminoglycosides, but not PTC124 (Ataluren), rescue nonsense mutations in the leptin receptor and in luciferase reporter genes.CFTR Modulators for the Treatment of Cystic Fibrosis.Aminoglycoside-Induced Premature Stop Codon Read-Through of Mucopolysaccharidosis Type I Patient Q70X and W402X Mutations in Cultured Cells.Dystrophins, utrophins, and associated scaffolding complexes: role in mammalian brain and implications for therapeutic strategies.Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosisTranslational read-through as an alternative approach for ocular gene therapy of retinal dystrophies caused by in-frame nonsense mutations.Targets for cystic fibrosis therapy: proteomic analysis and correction of mutant cystic fibrosis transmembrane conductance regulatorPharmacologic management of Duchenne muscular dystrophy: target identification and preclinical trialsThe effects of low levels of dystrophin on mouse muscle function and pathologyGene therapy for muscular dystrophies: progress and challengesToward a rationale for the PTC124 (Ataluren) promoted readthrough of premature stop codons: a computational approach and GFP-reporter cell-based assayAminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophyTranslational read-through of the RP2 Arg120stop mutation in patient iPSC-derived retinal pigment epithelium cells.Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression.Ataluren treatment of patients with nonsense mutation dystrophinopathy.Dystrophin quantification: Biological and translational research implications.Gene therapy for muscular dystrophy: moving the field forward.PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse modelA new series of small molecular weight compounds induce read through of all three types of nonsense mutations in the ATM gene.Making sense of nonsense GABA(A) receptor mutations associated with genetic epilepsies.In vivo readout of CFTR function: ratiometric measurement of CFTR-dependent secretion by individual, identifiable human sweat glandsChemical treatment enhances skipping of a mutated exon in the dystrophin gene.Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.Nonsense suppressor therapies rescue peroxisome lipid metabolism and assembly in cells from patients with specific PEX gene mutations
P2860
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P2860
Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.
description
2007 nî lūn-bûn
@nan
2007 թուականի Ապրիլին հրատարակուած գիտական յօդուած
@hyw
2007 թվականի ապրիլին հրատարակված գիտական հոդված
@hy
2007年の論文
@ja
2007年論文
@yue
2007年論文
@zh-hant
2007年論文
@zh-hk
2007年論文
@zh-mo
2007年論文
@zh-tw
2007年论文
@wuu
name
Safety, tolerability, and phar ...... e and female adult volunteers.
@ast
Safety, tolerability, and phar ...... e and female adult volunteers.
@en
Safety, tolerability, and phar ...... e and female adult volunteers.
@nl
type
label
Safety, tolerability, and phar ...... e and female adult volunteers.
@ast
Safety, tolerability, and phar ...... e and female adult volunteers.
@en
Safety, tolerability, and phar ...... e and female adult volunteers.
@nl
prefLabel
Safety, tolerability, and phar ...... e and female adult volunteers.
@ast
Safety, tolerability, and phar ...... e and female adult volunteers.
@en
Safety, tolerability, and phar ...... e and female adult volunteers.
@nl
P2093
P2860
P356
P1476
Safety, tolerability, and phar ...... e and female adult volunteers.
@en
P2093
Eileen M Leonard
Ellen M Welch
Gary L Elfring
Langdon L Miller
Neil G Almstead
Samit Hirawat
Seongwoo Hwang
Sergey Paushkin
Stuart W Peltz
Valerie J Northcutt
P2860
P304
P356
10.1177/0091270006297140
P577
2007-04-01T00:00:00Z