2025
Emerging autism and Fragile X syndrome treatments
Parkhill M, Salmaso N, D'Angiulli A, Lee V, Aguilar-Valles A. Emerging autism and Fragile X syndrome treatments. Trends In Pharmacological Sciences 2025 PMID: 40102109, DOI: 10.1016/j.tips.2025.02.004.Peer-Reviewed Original ResearchFragile X syndromeCore symptoms of autismSymptom-focused treatmentTreating core symptomsSymptoms of autismFragile X syndrome treatmentBiology of autismEndocannabinoid modulationCore symptomsDisorder heterogeneityX syndromeAutismPersonalized treatment approachesPharmacological interventionsTreatment approachesDisordersLate-phase trialsAnti-inflammatory approachesEndocannabinoidEffective therapyRecent trialsClinical trialsInterventionTrialsMicrobiome therapies
2024
Conditional Activation of c-MYC in Distinct Catecholaminergic Cells Drives Development of Neuroblastoma or Somatostatinoma
Wang T, Liu L, Fang J, Jin H, Natarajan S, Sheppard H, Lu M, Turner G, Confer T, Johnson M, Steinberg J, Ha L, Yadak N, Jain R, Picketts D, Ma X, Murphy A, Davidoff A, Glazer E, Easton J, Chen X, Wang R, Yang J. Conditional Activation of c-MYC in Distinct Catecholaminergic Cells Drives Development of Neuroblastoma or Somatostatinoma. Cancer Research 2024, 85: 424-441. PMID: 39531507, PMCID: PMC11786959, DOI: 10.1158/0008-5472.can-24-1142.Peer-Reviewed Original ResearchPancreatic neuroendocrine tumorsC-mycCre recombinaseAnti-GD2 immunotherapyHigh-risk neuroblastomaDopamine B-hydroxylaseActivity of c-MycFDA-approved inhibitorC-Myc overexpressionC-Myc activityC-myc inductionTesting immunotherapyNeuroendocrine tumorsTargeted therapyTumor typesNeuroblastoma developmentNeuroblastoma tumorsNeuroblastoma oncogenesisEffective therapyTyrosine hydroxylaseTarget cellsTumorNeuroblastomaHuman neuroblastomaGenetic featuresEarly vigabatrin to augment GABAergic pathways in post-anoxic status epilepticus
Maciel C, Ahmad B, Jose Bruzzone Giraldez M, Eisenschenk S, Ramsay E, Maranchick N, Peloquin C, Hirsch L, Busl K. Early vigabatrin to augment GABAergic pathways in post-anoxic status epilepticus. Epilepsy & Behavior 2024, 160: 110082. PMID: 39393141, DOI: 10.1016/j.yebeh.2024.110082.Peer-Reviewed Original ResearchStatus epilepticusGamma-aminobutyric acidGABAergic pathwayHypoxic-ischemic brain injuryPost-cardiac arrest periodHypoxic-ischemic insultOutcomes of patientsEffective adjunctive therapyInhibition of gamma-aminobutyric acidLondon-Innsbruck ColloquiumAdjunctive therapyAcute seizuresEffective therapyPoor outcomeEpilepticusExclusion criteriaEarly inhibitionSynergistic augmentationGamma-aminobutyric acid catabolismArrest periodTherapeutic nihilismSeizuresClinal trialsAllosteric modulatorsVigabatrinA brave new framework for glioma drug development
Hotchkiss K, Karschnia P, Schreck K, Geurts M, Cloughesy T, Huse J, Duke E, Lathia J, Ashley D, Nduom E, Long G, Singh K, Chalmers A, Ahluwalia M, Heimberger A, Bagley S, Todo T, Verhaak R, Kelly P, Hervey-Jumper S, de Groot J, Patel A, Fecci P, Parney I, Wykes V, Watts C, Burns T, Sanai N, Preusser M, Tonn J, Drummond K, Platten M, Das S, Tanner K, Vogelbaum M, Weller M, Whittle J, Berger M, Khasraw M. A brave new framework for glioma drug development. The Lancet Oncology 2024, 25: e512-e519. PMID: 39362262, DOI: 10.1016/s1470-2045(24)00190-6.Peer-Reviewed Original ResearchConceptsBrain tumorsBenefits of biopsyBrain tumor therapyLiquid biopsy technologiesTissue samplesPostoperative deficitsBiopsy techniqueBiopsy technologyEffective therapySurgical trialsClinical trialsTumor therapyResistance mechanismsTumorTherapyPatientsDrug developmentTissue analysisBrainTrialsTissueBiopsyGliomaRegulatory agenciesGastrointestinal disease in systemic sclerosis: the neglected organ system?
McMahan Z, Pandolfino J, Perlman H, Del Galdo F, Hinchcliff M. Gastrointestinal disease in systemic sclerosis: the neglected organ system? Current Opinion In Rheumatology 2024, 36: 374-378. PMID: 39193877, PMCID: PMC11588520, DOI: 10.1097/bor.0000000000001052.Peer-Reviewed Original ResearchSystemic sclerosisClinical trial endpointsInflammatory bowel diseaseTrial endpointsAssessment of disease activityRisk stratification methodsGastrointestinal (GIDisease activityRisk stratificationEffective therapyImmune responseBowel diseaseDisease pathogenesisTherapeutic interventionsGastrointestinal diseasesOrgan systemsDiseaseMultidisciplinary teamPathogenesisFunctional studiesGut microbiomeEndpointSclerosisSingle-cell multi-cohort dissection of the schizophrenia transcriptome
Ruzicka W, Mohammadi S, Fullard J, Davila-Velderrain J, Subburaju S, Tso D, Hourihan M, Jiang S, Lee H, Bendl J, Voloudakis G, Haroutunian V, Hoffman G, Roussos P, Kellis M, Akbarian S, Abyzov A, Ahituv N, Arasappan D, Almagro Armenteros J, Beliveau B, Berretta S, Bharadwaj R, Bhattacharya A, Bicks L, Brennand K, Capauto D, Champagne F, Chatterjee T, Chatzinakos C, Chen Y, Chen H, Cheng Y, Cheng L, Chess A, Chien J, Chu Z, Clarke D, Clement A, Collado-Torres L, Cooper G, Crawford G, Dai R, Daskalakis N, Deep-Soboslay A, Deng C, DiPietro C, Dracheva S, Drusinsky S, Duan Z, Duong D, Dursun C, Eagles N, Edelstein J, Emani P, Galani K, Galeev T, Gandal M, Gaynor S, Gerstein M, Geschwind D, Girdhar K, Goes F, Greenleaf W, Grundman J, Guo H, Guo Q, Gupta C, Hadas Y, Hallmayer J, Han X, Hawken N, He C, Henry E, Hicks S, Ho M, Ho L, Huang Y, Huuki-Myers L, Hwang A, Hyde T, Iatrou A, Inoue F, Jajoo A, Jensen M, Jiang L, Jin P, Jin T, Jops C, Jourdon A, Kawaguchi R, Kleinman J, Kleopoulos S, Kozlenkov A, Kriegstein A, Kundaje A, Kundu S, Lee C, Lee D, Li J, Li M, Lin X, Liu S, Liu J, Liu J, Liu C, Liu S, Lou S, Loupe J, Lu D, Ma S, Ma L, Margolis M, Mariani J, Martinowich K, Maynard K, Mazariegos S, Meng R, Myers R, Micallef C, Mikhailova T, Ming G, Monte E, Montgomery K, Moore J, Moran J, Mukamel E, Nairn A, Nemeroff C, Ni P, Norton S, Nowakowski T, Omberg L, Page S, Park S, Patowary A, Pattni R, Pertea G, Peters M, Phalke N, Pinto D, Pjanic M, Pochareddy S, Pollard K, Pollen A, Pratt H, Przytycki P, Purmann C, Qin Z, Qu P, Quintero D, Raj T, Rajagopalan A, Reach S, Reimonn T, Ressler K, Ross D, Rozowsky J, Ruth M, Sanders S, Schneider J, Scuderi S, Sebra R, Sestan N, Seyfried N, Shao Z, Shedd N, Shieh A, Shin J, Skarica M, Snijders C, Song H, State M, Stein J, Steyert M, Sudhof T, Snyder M, Tao R, Therrien K, Tsai L, Urban A, Vaccarino F, van Bakel H, Vo D, Wamsley B, Wang T, Wang S, Wang D, Wang Y, Warrell J, Wei Y, Weimer A, Weinberger D, Wen C, Weng Z, Whalen S, White K, Willsey A, Won H, Wong W, Wu H, Wu F, Wuchty S, Wylie D, Xu S, Yap C, Zeng B, Zhang P, Zhang C, Zhang B, Zhang J, Zhang Y, Zhou X, Ziffra R, Zeier Z, Zintel T. Single-cell multi-cohort dissection of the schizophrenia transcriptome. Science 2024, 384: eadg5136. PMID: 38781388, DOI: 10.1126/science.adg5136.Peer-Reviewed Original ResearchConceptsGenetic risk factorsRisk factorsTranscriptional changesHeterogeneity of schizophreniaNeuronal cell statesSchizophrenia pathophysiologySingle-cell dissectionExcitatory neuronsEffective therapySchizophrenia transcriptomicsCortical cytoarchitectureSingle-cell atlasGenomic variantsCell groupsHuman prefrontal cortexMolecular pathwaysSchizophreniaTranscriptional alterationsTranscriptomic changesPrefrontal cortexCell statesAlterationsTherapyPathophysiologyDissectionTANGO1 inhibitors reduce collagen secretion and limit tissue scarring
Raote I, Rosendahl A, Häkkinen H, Vibe C, Küçükaylak I, Sawant M, Keufgens L, Frommelt P, Halwas K, Broadbent K, Cunquero M, Castro G, Villemeur M, Nüchel J, Bornikoel A, Dam B, Zirmire R, Kiran R, Carolis C, Andilla J, Loza-Alvarez P, Ruprecht V, Jamora C, Campelo F, Krüger M, Hammerschmidt M, Eckes B, Neundorf I, Krieg T, Malhotra V. TANGO1 inhibitors reduce collagen secretion and limit tissue scarring. Nature Communications 2024, 15: 3302. PMID: 38658535, PMCID: PMC11043333, DOI: 10.1038/s41467-024-47004-1.Peer-Reviewed Original ResearchConceptsEndoplasmic reticulum exit sitesECM proteinsSecretion of ECM proteinsPeptide inhibitorFibrotic diseasesCollagen exportTANGO1Binding interfaceWound healingZebrafish resultsECM componentsReduced granulation tissue formationGranulation tissue formationEffective therapyCutaneous wound healingInhibitor treatmentFibrotic processUncontrolled secretionWidespread occurrenceProtein levelsExit siteExcessive scarringAmount of collagenTherapeutic modulationTissue scarringVaccines as Immunotherapies for Substance Use Disorders
Kosten T. Vaccines as Immunotherapies for Substance Use Disorders. American Journal Of Psychiatry 2024, 181: 362-371. PMID: 38706331, DOI: 10.1176/appi.ajp.20230828.Peer-Reviewed Original ResearchConceptsSubstance use disordersHuman clinical trials of vaccinesPreclinical vaccine studiesClinical trials of vaccinesVaccine clinical trialsHuman clinical trialsTrials of vaccinesVaccine immunotherapyIllicit fentanyl useOpioid pharmacotherapyEffective therapySubstance use disorder treatmentFentanyl useClinical trialsVaccine studiesAnimal modelsAnimal studiesUse disorderVaccine technologyLethal overdoseImmunotherapyVaccineLimited treatment accessFentanyl overdoseTreatment accessComplement protein signatures in patients with alcohol-associated hepatitis
Taiwo M, Huang E, Pathak V, Bellar A, Welch N, Dasarathy J, Streem D, McClain C, Mitchell M, Barton B, Szabo G, Dasarathy S, Consortium A, Schaefer E, Luther J, Day L, Ouyang X, Suyavaran A, Mehal W, Jacobs J, Goodman R, Rotroff D, Nagy L. Complement protein signatures in patients with alcohol-associated hepatitis. JCI Insight 2024, 9: e174127. PMID: 38573776, PMCID: PMC11141929, DOI: 10.1172/jci.insight.174127.Peer-Reviewed Original ResearchAlcohol-associated hepatitisSevere AHAlcohol use disorderAlcoholic cirrhosisHealthy controlsPredicting 90-day mortalityComplement proteinsSerum proteome of patientsEthanol-induced liver injurySerum proteomeDevelopment of effective therapiesProteome of patientsAssociated with pro-inflammatory cytokinesProtein signaturesPro-inflammatory cytokinesCoagulation factors IINon-invasive biomarkersDiagnostic challengeSerine protease 1Murine modelEffective therapyLiver injuryPrognostic biomarkerHepatic inflammationC1q binding proteinDapagliflozin and Timing of Prior Heart Failure Hospitalization A Patient-Level Meta-Analysis of DAPA-HF and DELIVER
Butt J, Jhund P, Docherty K, Claggett B, Vaduganathan M, Bachus E, Hernandez A, Lam C, Inzucchi S, Martinez F, de Boer R, Kosiborod M, Desai A, Køber L, Ponikowski P, Sabatine M, Solomon S, McMurray J. Dapagliflozin and Timing of Prior Heart Failure Hospitalization A Patient-Level Meta-Analysis of DAPA-HF and DELIVER. JACC Heart Failure 2024, 12: 1586-1599. PMID: 38573262, DOI: 10.1016/j.jchf.2024.01.018.Peer-Reviewed Original ResearchConceptsHF hospitalizationDAPA-HFHeart failureComposite of worsening HFPrimary outcomePatient-level pooled analysisRisk of adverse clinical outcomesBenefits of dapagliflozinMedian follow-upMonths of randomizationEffects of dapagliflozinAdverse clinical outcomesPrior HF hospitalizationHospital categoryPrimary endpointPrevent 1 eventClinical outcomesAbsolute benefitEffective therapyFollow-upCardiovascular deathDapagliflozinAssociated with timeHigh riskPatientsManagement of Incomplete Microcirculatory Reperfusion After Endovascular Thrombectomy: Focus on Inhibition of the Glycoprotein IIb/IIIa Receptor Pathway
Krothapalli N, Ortel T, McBride D, de Havenon A, Sansing L, Hasan D, Mac Grory B. Management of Incomplete Microcirculatory Reperfusion After Endovascular Thrombectomy: Focus on Inhibition of the Glycoprotein IIb/IIIa Receptor Pathway. Stroke Vascular And Interventional Neurology 2024, 4 DOI: 10.1161/svin.123.001048.Peer-Reviewed Original ResearchMicrocirculatory reperfusionEndovascular thrombectomyAdministration of tirofibanDigital subtraction angiographyLarge-vessel occlusionProportion of casesRescue therapyTherapeutic optionsAntiplatelet therapyClinical outcomesUnfavorable outcomeEndothelial edemaSubtraction angiographyEffective therapyIntraarterial administrationClinical challengeReceptor pathwayPlatelet aggregationTreatment approachesTherapyReperfusionCerebral microvasculatureTissue recoveryNeurological diseasesThrombectomyVagus nerve stimulation rescues persistent pain following orthopedic surgery in adult mice
Wu P, Caceres A, Chen J, Sokoloff J, Huang M, Baht G, Nackley A, Jordt S, Terrando N. Vagus nerve stimulation rescues persistent pain following orthopedic surgery in adult mice. Pain 2024, 165: e80-e92. PMID: 38422485, PMCID: PMC11247455, DOI: 10.1097/j.pain.0000000000003181.Peer-Reviewed Original ResearchDorsal root gangliaPostoperative painVagus nerve stimulationPain behaviorOrthopedic surgeryNerve stimulationHindpaw mechanical allodyniaPrevent postoperative painFemale C57BL/6J miceOrthopedic trauma surgeryMechanical allodyniaAntinociceptive effectPersistent painAccelerated bone healingAnalgesic effectAdult miceC57BL/6J miceEffective therapyNeuropeptide levelsSpinal cordMouse modelSatellite cellsSurgeryPainVagus nerveReal-World Tralokinumab Use in Dupilumab-Experienced Patients: A Eetrospective Multi-Center Case Series
Herman E, Burgy J, Shahriari M. Real-World Tralokinumab Use in Dupilumab-Experienced Patients: A Eetrospective Multi-Center Case Series. SKIN The Journal Of Cutaneous Medicine 2024, 8: s321. DOI: 10.25251/skin.8.supp.321.Peer-Reviewed Original ResearchTreated with dupilumabAtopic dermatitisDupilumab treatmentTralokinumab treatmentCase seriesSafety profileLong-term disease controlHead-to-head studiesDiscontinued dupilumab treatmentTreated with topicalMulti-center case seriesDuration of treatmentLack of efficacyEvaluate clinical findingsBiologic dupilumabHerpes labialisNRS scoresAdult patientsClinical findingsDupilumabTreatment optionsEffective therapyTralokinumabDisease durationJoint pain
2023
Are We Ready For “Triplet” Therapy in Higher-Risk MDS?
Brunner A, Platzbecker U, DeZern A, Zeidan A. Are We Ready For “Triplet” Therapy in Higher-Risk MDS? Clinical Hematology International 2023, 5: 23-32. PMID: 37933301, PMCID: PMC10625655, DOI: 10.46989/001c.88301.Peer-Reviewed Original ResearchAcute myeloid leukemiaCombination chemotherapyPatient populationOnly disease-modifying therapyNovel combination chemotherapyOngoing therapeutic challengeOlder patient populationUnique patient populationDisease-modifying therapiesCurrent treatment paradigmsTherapeutic challengeTreatment paradigmEffective therapyMyeloid leukemiaNew therapiesAppropriate endpointsTherapyChemotherapyComorbiditiesPatientsTransplantNeoplasmsLeukemiaPopulationDecitabineIntrathecal delivery of nanoparticle PARP inhibitor to the cerebrospinal fluid for the treatment of metastatic medulloblastoma
Khang M, Lee J, Lee T, Suh H, Lee S, Cavaliere A, Rushing A, Geraldo L, Belitzky E, Rossano S, de Feyter H, Shin K, Huttner A, Roussel M, Thomas J, Carson R, Marquez-Nostra B, Bindra R, Saltzman W. Intrathecal delivery of nanoparticle PARP inhibitor to the cerebrospinal fluid for the treatment of metastatic medulloblastoma. Science Translational Medicine 2023, 15: eadi1617. PMID: 37910601, PMCID: PMC11078331, DOI: 10.1126/scitranslmed.adi1617.Peer-Reviewed Original ResearchConceptsCerebrospinal fluidDelivery of drugsEffective therapyTherapeutic indexPARP inhibitorsBlood-brain barrierSite of tumorRapid systemic clearanceXenograft mouse modelSolvent evaporation processAdministration of substancesLeptomeningeal spreadIntrathecal deliveryLeptomeningeal metastasesBrain penetrationSystemic clearanceTumor regressionPolymer nanoparticlesMetastatic medulloblastomaMouse modelPediatric medulloblastomaDrug accumulationCSF turnoverEncapsulated drugsPET imagingPhototherapy Restores Deficient Type I IFN Production and Enhances Antitumor Responses in Mycosis Fungoides
Yu Z, Vieyra-Garcia P, Benezeder T, Crouch J, Kim I, O'Malley J, Devlin P, Gehad A, Zhan Q, Gudjonsson J, Sarkar M, Kahlenberg J, Gerard N, Teague J, Kupper T, LeBoeuf N, Larocca C, Tawa M, Pomahac B, Talbot S, Orgill D, Wolf P, Clark R. Phototherapy Restores Deficient Type I IFN Production and Enhances Antitumor Responses in Mycosis Fungoides. Journal Of Investigative Dermatology 2023, 144: 621-632.e1. PMID: 37716650, PMCID: PMC10922223, DOI: 10.1016/j.jid.2023.06.212.Peer-Reviewed Original ResearchType I IFNType I IFN productionI IFN expressionI IFN productionI IFNUVA therapyAntitumor responseSkin lesionsIFN productionIFN expressionMycosis fungoides skin lesionsType I IFN expressionAntigen-specific T cell activationEffective tumor clearanceType I interferon productionEnhanced antitumor responseSun-protected skinIFN response genesI interferon productionT cell activationType I IFN gene expressionIFN gene expressionTumor clearanceMycosis fungoidesEffective therapyIdentifying factors that influence the decision to reduce, delay, or discontinue treatment due to chemotherapy-induced peripheral neuropathy: A community-centered approach.
Radwan R, Hertz D, Hickey E, Vachhani H, Lustberg M, Bridges J, Sabo R, Sheppard V, Salgado T. Identifying factors that influence the decision to reduce, delay, or discontinue treatment due to chemotherapy-induced peripheral neuropathy: A community-centered approach. Journal Of Clinical Oncology 2023, 41: e13123-e13123. DOI: 10.1200/jco.2023.41.16_suppl.e13123.Peer-Reviewed Original ResearchChemotherapy-induced peripheral neuropathyMetastatic breast cancerPeripheral neuropathyTreatment alterationsTreatment effectivenessMBC patientsCIPN symptomsMedical oncologistsDiscontinue treatmentClinical guidelinesOncology cliniciansCommunity-centered approachEffective therapyBreast cancerChemotherapy agentsTreatment decisionsPatient prioritiesPatient's perspectiveDose reductionAlternative treatmentOptimize outcomesPatientsSide effectsTreatment planHealthcare providersFrailty Resilience Score: A Novel Measure of Frailty Resilience Associated With Protection From Frailty and Survival
Milman S, Lerman B, Ayers E, Zhang Z, Sathyan S, Levine M, Ye K, Gao T, Higgins-Chen A, Barzilai N, Verghese J. Frailty Resilience Score: A Novel Measure of Frailty Resilience Associated With Protection From Frailty and Survival. The Journals Of Gerontology Series A 2023, 78: 1771-1777. PMID: 37246648, PMCID: PMC10562888, DOI: 10.1093/gerona/glad138.Peer-Reviewed Original ResearchConceptsMultivariable-adjusted analysesHazard of mortalityPhenotypic frailtyBaseline frailtyOverall survivalEffective therapyHigh riskFrailtyResilience AssociatedOlder adultsGenetic riskIdentification of factorsReliable predictorMortalityResilience scoresSurvivalProteomic profilesRiskReliable measureDeviation increaseTherapyCohortRecent Advances in the Management of Primary Sclerosing Cholangitis
Assis D, Bowlus C. Recent Advances in the Management of Primary Sclerosing Cholangitis. Clinical Gastroenterology And Hepatology 2023, 21: 2065-2075. PMID: 37084929, DOI: 10.1016/j.cgh.2023.04.004.Peer-Reviewed Original ResearchConceptsChronic cholestatic liver diseasePrimary sclerosing cholangitisInflammatory bowel diseasePrognostication of patientsCholestatic liver diseaseSclerosing cholangitisBowel diseaseLiver failureClinical featuresLiver diseaseMedical managementBiliary treeComplex pathophysiologyEffective therapyPharmacologic agentsRare natureCurrent conceptsDiseaseCholangitisFurther studiesCholangiocarcinomaPatientsPathophysiologyTherapyPrognosticationVagus Nerve Stimulation Rescues Persistent Pain Following Orthopedic Surgery In Adult Mice
Wu P, De Caceres A, Baht G, Terrando N, Jordt S. Vagus Nerve Stimulation Rescues Persistent Pain Following Orthopedic Surgery In Adult Mice. Journal Of Pain 2023, 24: 10. DOI: 10.1016/j.jpain.2023.02.044.Peer-Reviewed Original ResearchVagus nerve stimulationPostoperative painOrthopedic surgeryNerve stimulationPain 2 yearsAnti-nociceptive effectsPersistent postoperative painSatellite glial cellsAnti-inflammatory effectsMajor clinical problemBilateral hindTibial surgeryMicroglia activationRisk patientsPain sensitizationPersistent painVagus nervePain behaviorEffective therapyFemale miceSpinal cordWeekly treatmentGlial cellsGFAP expressionInvasive procedures
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