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ataluren

Known as: EC-000.2051 
National Institutes of Health

Related topics

Related topics

2 relations

Broader (1)

Oxadiazoles

Narrower (1)

PTC 124

Papers overview

Semantic Scholar uses AI to extract papers important to this topic.
Review
2015
Review
2015
European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene
Highly Cited
2014
Highly Cited
2014
Ataluren treatment of patients with nonsense mutation dystrophinopathy
Introduction: Dystrophinopathy is a rare, severe muscle disorder, and nonsense mutations are found in 13% of cases. Ataluren was… 
Highly Cited
2014
Highly Cited
2014
Toward a Rationale for the PTC124 (Ataluren) Promoted Readthrough of Premature Stop Codons: A Computational Approach and GFP-Reporter Cell-Based Assay
The presence in the mRNA of premature stop codons (PTCs) results in protein truncation responsible for several inherited (genetic… 
Highly Cited
2014
Highly Cited
2014
Muscle dysfunction and structural defects of dystrophin‐null sapje mutant zebrafish larvae are rescued by ataluren treatment
Sapje zebrafish carry a mutation in the dystrophin gene, which results in a premature stop codon, and a severe muscle phenotype… 
Highly Cited
2013
Highly Cited
2013
Correction of nonsense BMPR2 and SMAD9 mutations by ataluren in pulmonary arterial hypertension.
Heritable pulmonary arterial hypertension (HPAH) is a serious lung vascular disease caused by heterozygous mutations in the bone… 
Highly Cited
2013
Highly Cited
2013
THE 6-MINUTE WALK TEST AND OTHER ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: LONGITUDINAL NATURAL HISTORY OBSERVATIONS OVER 48 WEEKS FROM A MULTICENTER STUDY
Introduction: Duchenne muscular dystrophy (DMD) subjects ≥5 years with nonsense mutations were followed for 48 weeks in a… 
Highly Cited
2013
Highly Cited
2013
A Lack of Premature Termination Codon Read-Through Efficacy of PTC124 (Ataluren) in a Diverse Array of Reporter Assays
The drug molecule PTC124 (Ataluren) has been described as a read-through agent, capable of suppressing premature termination… 
Review
2013
Review
2013
Development, clinical utility, and place of ivacaftor in the treatment of cystic fibrosis
Cystic fibrosis (CF) is a life-limiting, multisystem disease characterized by thick viscous secretions leading to recurrent lung… 
Highly Cited
2010
Highly Cited
2010
Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124
Firefly luciferase (FLuc), an ATP-dependent bioluminescent reporter enzyme, is broadly used in chemical biology and drug… 
2009
2009
Nonsense suppression activity of PTC124 (ataluren)
Auld et al. (1) suggest that PTC124's nonsense suppression activity may be an indirect consequence of the compound's effects on… 
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