Found: 33
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In vitro metabolism, reaction phenotyping, enzyme kinetics, CYP inhibition and induction potential of ataluren.
- Published in:
- Pharmacology Research & Perspectives, 2020, v. 8, n. 2, p. 1, doi. 10.1002/prp2.576
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- Publication type:
- Article
Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs.
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- Pharmacology Research & Perspectives, 2018, v. 6, n. 6, p. 1, doi. 10.1002/prp2.447
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- Publication type:
- Article
Pharmacokinetics and Safety of PTC596, a Novel Tubulin‐Binding Agent, in Subjects With Advanced Solid Tumors.
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- Clinical Pharmacology in Drug Development, 2021, v. 10, n. 8, p. 940, doi. 10.1002/cpdd.904
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- Publication type:
- Article
Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress-Regulated Protein Translation.
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- Clinical Pharmacology in Drug Development, 2016, v. 5, n. 4, p. 296, doi. 10.1002/cpdd.240
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- Publication type:
- Article
Editorial: New therapeutics for soft tissue sarcomas.
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- Frontiers in Oncology, 2023, p. 1, doi. 10.3389/fonc.2023.1297215
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- Publication type:
- Article
Small molecule splicing modifiers with systemic HTT-lowering activity.
- Published in:
- Nature Communications, 2021, v. 12, n. 1, p. 1, doi. 10.1038/s41467-021-27157-z
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- Publication type:
- Article
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.
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- British Journal of Haematology, 2020, v. 190, n. 6, p. 877, doi. 10.1111/bjh.16595
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- Article
Absorption and disposition of a tripeptoid and a tetrapeptide in the rat.
- Published in:
- Biopharmaceutics & Drug Disposition, 1999, v. 20, n. 2, p. 69, doi. 10.1002/(SICI)1099-081X(199903)20:2<69::AID-BDD153>3.0.CO;2-B
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- Publication type:
- Article
Discovery and SAR Studies of Potent Modulators of BMI‐1 Expression.
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- Helvetica Chimica Acta, 2023, v. 106, n. 9, p. 1, doi. 10.1002/hlca.202300076
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- Publication type:
- Article
Emvododstat, a Potent Dihydroorotate Dehydrogenase Inhibitor, Is Effective in Preclinical Models of Acute Myeloid Leukemia.
- Published in:
- Frontiers in Oncology, 2022, v. 12, p. 1, doi. 10.3389/fonc.2022.832816
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- Publication type:
- Article
THER-14. SMALL MOLECULE INHIBITOR TARGETING SELF-RENEWAL AS A THERAPEUTIC OPTION FOR RECURRENT MEDULLOBLASTOMA.
- Published in:
- Neuro-Oncology, 2019, v. 21, p. ii117, doi. 10.1093/neuonc/noz036.221
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- Publication type:
- Article
THER-11. BMI-1 MODULATION IMPAIRS DNA DAMAGE RESPONSE AND SENSITIZES DIPG CELLS TO IONIZING RADIATION.
- Published in:
- Neuro-Oncology, 2019, v. 21, p. ii116, doi. 10.1093/neuonc/noz036.218
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- Publication type:
- Article
ATRT-05. PRC1 IS AN ESSENTIAL DEPENDENCY AND THERAPEUTIC TARGET IN SMARCB1 DEFICIENT ATYPICAL TERATOID RHABDOID TUMORS.
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- Neuro-Oncology, 2019, v. 21, p. ii63, doi. 10.1093/neuonc/noz036.004
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- Publication type:
- Article
Improved binding of a bivalent single-chain immunotoxin results in increased efficacy for in vivo T-cell depletion.
- Published in:
- Protein Engineering, 2001, v. 14, n. 12, p. 1035, doi. 10.1093/protein/14.12.1035
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- Publication type:
- Article
BMI1 as a novel target for drug discovery in cancer.
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- Journal of Cellular Biochemistry, 2011, v. 112, n. 10, p. 2729, doi. 10.1002/jcb.23234
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- Publication type:
- Article
AML-284: Emvododstat (PTC299) Targets De Novo Pyrimidine Synthesis in Acute Myeloid Leukemia.
- Published in:
- Clinical Lymphoma, Myeloma & Leukemia, 2021, v. 21, p. S300, doi. 10.1016/S2152-2650(21)01714-6
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- Publication type:
- Article
Poster: AML-284: Emvododstat (PTC299) Targets De Novo Pyrimidine Synthesis in Acute Myeloid Leukemia.
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- Clinical Lymphoma, Myeloma & Leukemia, 2021, v. 21, p. S215, doi. 10.1016/S2152-2650(21)01356-2
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- Publication type:
- Article
Targeting of BMI-1 in Mantle Cell Lymphoma.
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- Clinical Lymphoma, Myeloma & Leukemia, 2018, v. 18, p. S275, doi. 10.1016/j.clml.2018.07.202
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- Publication type:
- Article
Targeting ferroptosis with the lipoxygenase inhibitor PTC-041 as a therapeutic strategy for the treatment of Parkinson's disease.
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- PLoS ONE, 2024, v. 19, n. 9, p. 1, doi. 10.1371/journal.pone.0309893
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- Publication type:
- Article
SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.
- Published in:
- Human Molecular Genetics, 2022, v. 31, n. 1, p. 82, doi. 10.1093/hmg/ddab220
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- Publication type:
- Article
Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
- Published in:
- Human Molecular Genetics, 2021, v. 30, n. 9, p. 843, doi. 10.1093/hmg/ddaa247
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- Publication type:
- Article
Functional rescue of REP1 following treatment with PTC124 and novel derivative PTC-414 in human choroideremia fibroblasts and the nonsense-mediated zebrafish model.
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- Human Molecular Genetics, 2016, v. 25, n. 16, p. 3416, doi. 10.1093/hmg/ddw184
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- Publication type:
- Article
Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. 5, p. 964, doi. 10.1093/hmg/ddv629
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- Publication type:
- Article
Discovery of Novel Small Molecule Inhibitors of VEGF Expression in Tumor Cells Using a Cell-Based High Throughput Screening Platform.
- Published in:
- PLoS ONE, 2016, v. 11, n. 12, p. 1, doi. 10.1371/journal.pone.0168366
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- Publication type:
- Article
SMN Protein Can Be Reliably Measured in Whole Blood with an Electrochemiluminescence (ECL) Immunoassay: Implications for Clinical Trials.
- Published in:
- PLoS ONE, 2016, v. 11, n. 3, p. 1, doi. 10.1371/journal.pone.0150640
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- Publication type:
- Article
Divergent pathways of gene expression are activated by the RAGE ligands S100b and AGE-BSA.
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- 2004
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- Publication type:
- journal article
Combinatorial approaches to inhibitors of VLA-4: Piperazine-peptoid-bisarylureas.
- Published in:
- Molecular Diversity, 2000, v. 5, n. 2, p. 61, doi. 10.1023/A:1013990624215
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- Publication type:
- Article
Ataluren suppresses a premature termination codon in an MPS I-H mouse.
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- Journal of Molecular Medicine, 2022, v. 100, n. 8, p. 1223, doi. 10.1007/s00109-022-02232-0
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- Publication type:
- Article
Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA.
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- Science Translational Medicine, 2021, v. 13, n. 578, p. 1, doi. 10.1126/scitranslmed.abb6871
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- Publication type:
- Article
The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential.
- Published in:
- PLoS ONE, 2018, v. 13, n. 10, p. 1, doi. 10.1371/journal.pone.0206158
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- Publication type:
- Article
Efficacy of the novel tubulin polymerization inhibitor PTC‐028 for myelodysplastic syndrome.
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- Cancer Science, 2020, v. 111, n. 12, p. 4336, doi. 10.1111/cas.14684
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- Publication type:
- Article
Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators.
- Published in:
- Scientific Reports, 2023, v. 13, n. 1, p. 1, doi. 10.1038/s41598-023-45376-w
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- Publication type:
- Article
Identification of benzazole compounds that induce HIV-1 transcription.
- Published in:
- PLoS ONE, 2017, v. 12, n. 6, p. 1, doi. 10.1371/journal.pone.0179100
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- Publication type:
- Article