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.
Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia.
Am J Hum Genet
; 110(3): 531-547, 2023 03 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36809767
3.
SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.
Hum Mol Genet
; 31(1): 82-96, 2021 12 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-34368854
4.
ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia.
Am J Hum Genet
; 104(4): 638-650, 2019 04 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-30905397
5.
Quantitation of Pax-6 protein in ocular impression cytology samples using an electrochemiluminescence immunoassay.
Anal Biochem
; 656: 114876, 2022 11 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36058293
6.
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
7.
Report of the National Institutes of Health SARS-CoV-2 Antiviral Therapeutics Summit.
J Infect Dis
; 224(Supplement_1): S1-S21, 2021 Jul 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-34111271
8.
Efficacy of the novel tubulin polymerization inhibitor PTC-028 for myelodysplastic syndrome.
Cancer Sci
; 111(12): 4336-4347, 2020 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-33037737
9.
The anti-mitotic agents PTC-028 and PTC596 display potent activity in pre-clinical models of multiple myeloma but challenge the role of BMI-1 as an essential tumour gene.
Br J Haematol
; 190(6): 877-890, 2020 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32232850
10.
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
11.
Functional rescue of REP1 following treatment with PTC124 and novel derivative PTC-414 in human choroideremia fibroblasts and the nonsense-mediated zebrafish model.
Hum Mol Genet
; 25(16): 3416-3431, 2016 08 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-27329764
12.
Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
Hum Mol Genet
; 25(5): 964-75, 2016 Mar 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-26758873
13.
Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
Hum Mol Genet
; 30(9): 843, 2021 May 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-33201218
14.
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
Hum Mol Genet
; 25(10): 1885-1899, 2016 05 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26931466
15.
4-Hydroxy-2-pyridones: Discovery and evaluation of a novel class of antibacterial agents targeting DNA synthesis.
Bioorg Med Chem Lett
; 27(22): 5014-5021, 2017 11 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-29032026
16.
Discovery of 2-(4-sulfonamidophenyl)-indole 3-carboxamides as potent and selective inhibitors with broad hepatitis C virus genotype activity targeting HCV NS4B.
Bioorg Med Chem Lett
; 26(2): 594-601, 2016 Jan 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26652483
17.
6-(Azaindol-2-yl)pyridine-3-sulfonamides as potent and selective inhibitors targeting hepatitis C virus NS4B.
Bioorg Med Chem Lett
; 25(4): 781-6, 2015 Feb 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-25613678
18.
Phase Ib Study of Unesbulin (PTC596) Plus Dacarbazine for the Treatment of Locally Recurrent, Unresectable or Metastatic, Relapsed or Refractory Leiomyosarcoma.
J Clin Oncol
; : JCO2301684, 2024 Apr 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-38684039
19.
Tumor Microenvironment Landscapes Supporting EGFR-mutant NSCLC Are Modulated at the Single-cell Interaction Level by Unesbulin Treatment.
Cancer Res Commun
; 4(3): 919-937, 2024 Mar 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-38546390
20.
Identification of PTC725, an orally bioavailable small molecule that selectively targets the hepatitis C Virus NS4B protein.
Antimicrob Agents Chemother
; 57(7): 3250-61, 2013 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-23629699