|
| 37293016 | Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators. | | 2023 |
| 37819629 | Comparison of pharmaceutical properties and biological activity of prednisolone, deflazacort, and vamorolone in DMD disease models. | | 2023 |
| 37909024 | Editorial: New therapeutics for soft tissue sarcomas. | | 2023 |
| 37903840 | Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators. | | 2023 |
| 36809767 | Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia. | | 2023 |
| 35223511 | Emvododstat, a Potent Dihydroorotate Dehydrogenase Inhibitor, Is Effective in Preclinical Models of Acute Myeloid Leukemia. | Frontiers in Oncology | 2022 |
| 36242990 | SAR studies toward discovery of emvododstat (PTC299), a potent dihydroorotate dehydrogenase (DHODH) inhibitor. | European Journal of Medicinal Chemistry | 2022 |
| 36157476 | Targeting BMI1 mitigates chemoresistance in ovarian cancer. | Genes and Diseases | 2022 |
| 36058293 | Quantitation of Pax-6 protein in ocular impression cytology samples using an electrochemiluminescence immunoassay. | Analytical Biochemistry | 2022 |
| 35857082 | Ataluren suppresses a premature termination codon in an MPS I-H mouse. | Journal of Molecular Medicine | 2022 |
| 34846990 | In vitro metabolism, pharmacokinetics and drug interaction potentials of emvododstat, a DHODH inhibitor. | Xenobiotica | 2022 |
| 34368854 | SMN protein is required throughout life to prevent spinal muscular atrophy disease progression. | Human Molecular Genetics | 2021 |
| 33483574 | The combination of the tubulin binding small molecule PTC596 and proteasome inhibitors suppresses the growth of myeloma cells. | Scientific Reports | 2021 |
| 33854168 | Identification of a targetable KRAS-mutant epithelial population in non-small cell lung cancer. | Commun Biol | 2021 |
| 34111271 | Report of the National Institutes of Health SARS-CoV-2 Antiviral Therapeutics Summit. | J Infect Dis | 2021 |
| 33504650 | Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA. | Sci Transl Med | 2021 |
| 33872944 | Ataluren metabolism: Ataluren-O-1β-acyl glucuronide is a stable circulating metabolite in mouse, rat, dog and human. | | 2021 |
| 33440067 | Pharmacokinetics and Safety of PTC596, a Novel Tubulin-Binding Agent, in Subjects With Advanced Solid Tumors. | Clin Pharmacol Drug Dev | 2021 |
| 34315764 | Preclinical and Early Clinical Development of PTC596, a Novel Small-Molecule Tubulin-Binding Agent. | Molecular Cancer Therapeutics | 2021 |
| 33496740 | DHODH inhibition synergizes with DNA-demethylating agents in the treatment of myelodysplastic syndromes. | Blood Adv | 2021 |
| 33201218 | Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset. | Human Molecular Genetics | 2021 |
| 33249060 | The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokines. | Virus Research | 2021 |
| 34911927 | Small molecule splicing modifiers with systemic HTT-lowering activity. | Nature Communications | 2021 |
| 32658832 | Chemical modifications of G418 (geneticin): Synthesis of novel readthrough aminoglycosides results in an improved in vitro safety window but no improvements in vivo. | Carbohydrate Research | 2020 |
| 32196986 | In vitro metabolism, reaction phenotyping, enzyme kinetics, CYP inhibition and induction potential of ataluren. | Pharmacology Research and Perspectives | 2020 |
| 32232850 | 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. | British Journal of Haematology | 2020 |
| 33037737 | Efficacy of the novel tubulin polymerization inhibitor PTC-028 for myelodysplastic syndrome. | Cancer Sci | 2020 |
| 32793904 | The DHODH Inhibitor PTC299 Arrests SARS-CoV-2 Replication and Suppresses Induction of Inflammatory Cytokines. | | 2020 |
| 30905397 | ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia. | American Journal of Human Genetics | 2019 |
| 30352802 | Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties. | Molecular Cancer Therapeutics | 2019 |
| 30348991 | BMI1 is a therapeutic target in recurrent medulloblastoma. | Oncogene | 2019 |
| 31175095 | Effective Delivery of a Microtubule Polymerization Inhibitor Synergizes with Standard Regimens in Models of Pancreatic Ductal Adenocarcinoma. | Clinical Cancer Research | 2019 |
| 29727185 | Discovery and Optimization of Indolyl-Containing 4-Hydroxy-2-Pyridone Type II DNA Topoisomerase Inhibitors Active against Multidrug Resistant Gram-negative Bacteria. | Journal of Medicinal Chemistry | 2018 |
| 29983879 | Targeting of BMI-1 expression by the novel small molecule PTC596 in mantle cell lymphoma. | Oncotarget | 2018 |
| 30359426 | The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential. | PLoS ONE | 2018 |
| 30026381 | Inhibition of BMI1, a Therapeutic Approach in Endometrial Cancer. | Molecular Cancer Therapeutics | 2018 |
| 30351051 | Correction to Discovery and Optimization of Indolyl-Containing 4-Hydroxy-2-pyridone Type II DNA Topoisomerase Inhibitors Active against Multidrug Resistant Gram-Negative Bacteria. | Journal of Medicinal Chemistry | 2018 |
| 30044619 | Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA). | J Med Chem | 2018 |
| 30519476 | Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. | Pharmacology Research and Perspectives | 2018 |
| 28096517 | The nucleoside analog clitocine is a potent and efficacious readthrough agent. | RNA | 2017 |
| 28658263 | Identification of benzazole compounds that induce HIV-1 transcription. | PLoS ONE | 2017 |
| 28441483 | Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy. | Journal of Medicinal Chemistry | 2017 |
| 29032026 | 4-Hydroxy-2-pyridones: Discovery and evaluation of a novel class of antibacterial agents targeting DNA synthesis. | Bioorganic and Medicinal Chemistry Letters | 2017 |
| 28211885 | The novel BMI-1 inhibitor PTC596 downregulates MCL-1 and induces p53-independent mitochondrial apoptosis in acute myeloid leukemia progenitor cells. | Blood Cancer Journal | 2017 |
| 27992500 | Discovery of Novel Small Molecule Inhibitors of VEGF Expression in Tumor Cells Using a Cell-Based High Throughput Screening Platform. | PLoS ONE | 2016 |
| 26758873 | Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset. | Human Molecular Genetics | 2016 |
| 27299569 | Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy. | Journal of Medicinal Chemistry | 2016 |
| 27299419 | Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy. | Journal of Medicinal Chemistry | 2016 |
| 26931466 | Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy. | Human Molecular Genetics | 2016 |
| 27329764 | 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 | 2016 |