(Q35240536)

7 August 2017

1 reference

title

Ataluren: first global approval (English)

1 reference

7 August 2017

Nicola J Ryan

1

1 reference

7 August 2017

1 September 2014

1 reference

7 August 2017

1 reference

7 August 2017

74

1 reference

7 August 2017

14

1 reference

7 August 2017

1709-1714

1 reference

https://scigraph.springernature.com/pub.10.1007/s40265-014-0287-4

7 August 2017

0 references

cites work

Translational readthrough induction of pathogenic nonsense mutations

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

When the message goes awry: disease-producing mutations that influence mRNA content and performance.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

PTC124 targets genetic disorders caused by nonsense mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

PTC124-mediated translational readthrough of a nonsense mutation causing Usher syndrome type 1C.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

Stop codon read-through with PTC124 induces palmitoyl-protein thioesterase-1 activity, reduces thioester load and suppresses apoptosis in cultured cells from INCL patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

Ataluren treatment of patients with nonsense mutation dystrophinopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40265-014-0287-4

PTC124 improves readthrough and increases enzymatic activity of the CPT1A R160X nonsense mutation

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25193627

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Muscle dysfunction and structural defects of dystrophin-null sapje mutant zebrafish larvae are rescued by ataluren treatment

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25193627

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25193627

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1007/S40265-014-0287-4

1 reference

7 August 2017

1 reference

PubMed ID

25193627