Detalhe da pesquisa
1.
Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models.
Hum Mol Genet
; 33(3): 211-223, 2024 Jan 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-37819629
2.
Guanidino quinazolines and pyrimidines promote readthrough of premature termination codons in cells with native nonsense mutations.
Bioorg Med Chem Lett
; 76: 128989, 2022 11 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-36150638
3.
In vitro metabolism, pharmacokinetics and drug interaction potentials of emvododstat, a DHODH inhibitor.
Xenobiotica
; 52(2): 152-164, 2022 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-34846990
4.
The nucleoside analog clitocine is a potent and efficacious readthrough agent.
RNA
; 23(4): 567-577, 2017 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-28096517
5.
Unexpected Observation of the Dimroth Rearrangement in the Ribosylation of 4-Aminopyrimidines.
J Org Chem
; 82(11): 5881-5889, 2017 06 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-28493695
6.
PTC124 targets genetic disorders caused by nonsense mutations.
Nature
; 447(7140): 87-91, 2007 May 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-17450125
7.
SAR studies toward discovery of emvododstat (PTC299), a potent dihydroorotate dehydrogenase (DHODH) inhibitor.
Eur J Med Chem
; 244: 114826, 2022 Dec 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-36242990
8.
Ataluren metabolism: Ataluren-O-1ß-acyl glucuronide is a stable circulating metabolite in mouse, rat, dog and human.
Drug Metab Pharmacokinet
; 38: 100393, 2021 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-33872944
9.
Preclinical and Early Clinical Development of PTC596, a Novel Small-Molecule Tubulin-Binding Agent.
Mol Cancer Ther
; 20(10): 1846-1857, 2021 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34315764
10.
Small molecule splicing modifiers with systemic HTT-lowering activity.
Nat Commun
; 12(1): 7299, 2021 12 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-34911927
11.
LC-MS/MS quantification of ataluren and ataluren acyl glucuronide in human plasma/urine: application in clinical studies.
Bioanalysis
; 12(21): 1545-1555, 2020 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-33064028
12.
Chemical modifications of G418 (geneticin): Synthesis of novel readthrough aminoglycosides results in an improved in vitro safety window but no improvements in vivo.
Carbohydr Res
; 495: 108058, 2020 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-32658832
13.
In vitro metabolism, reaction phenotyping, enzyme kinetics, CYP inhibition and induction potential of ataluren.
Pharmacol Res Perspect
; 8(2): e00576, 2020 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-32196986
14.
Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties.
Mol Cancer Ther
; 18(1): 3-16, 2019 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-30352802
15.
BMI1 is a therapeutic target in recurrent medulloblastoma.
Oncogene
; 38(10): 1702-1716, 2019 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30348991
16.
Evaluating the Mechanism and Therapeutic Potential of PTC-028, a Novel Inhibitor of BMI-1 Function in Ovarian Cancer.
Mol Cancer Ther
; 17(1): 39-49, 2018 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29158468
17.
The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential.
PLoS One
; 13(10): e0206158, 2018.
Artigo
em Inglês
| MEDLINE | ID: mdl-30359426
18.
Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress-Regulated Protein Translation.
Clin Pharmacol Drug Dev
; 5(4): 296-305, 2016 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-27310330
19.
BMI-1 Targeting Interferes with Patient-Derived Tumor-Initiating Cell Survival and Tumor Growth in Prostate Cancer.
Clin Cancer Res
; 22(24): 6176-6191, 2016 Dec 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-27307599
20.
Discovery of Novel Small Molecule Inhibitors of VEGF Expression in Tumor Cells Using a Cell-Based High Throughput Screening Platform.
PLoS One
; 11(12): e0168366, 2016.
Artigo
em Inglês
| MEDLINE | ID: mdl-27992500