2025
Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus
Oskoui M, Caller T, Parsons J, Servais L, Butterfield R, Bharadwaj J, Rose S, Tolchin B, Puskala Hamel K, Silsbee H, Dowling J. Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus. Neurology 2025, 104: e213604. PMID: 40367405, DOI: 10.1212/wnl.0000000000213604.Peer-Reviewed Original ResearchConceptsDuchenne muscular dystrophyFood and Drug AdministrationClinical trialsImmune-related side effectsMuscular dystrophyFood and Drug Administration approvalFunctional motor outcomePhase 3 clinical trialsTreatment groupsNorth Star Ambulatory Assessment scoresUS Food and Drug AdministrationMotor outcomeClinical trial dataRisk of biasClass I studiesGene therapyTreated patientsAmerican Academy of NeurologyLiver toxicityPrimary outcomeLiver injurySide effectsDrug AdministrationSafety outcomesDose exposureA plain language summary of the ASCERTAIN trial: oral decitabine and cedazuridine versus intravenous decitabine for MDS or CMML
Garcia-Manero G, McCloskey J, Griffiths E, Yee K, Zeidan A, Al-Kali A, Deeg H, Sabloff M, Keating M, Zhu N, Gabrail N, Fazal S, Maly J, Odenike O, DeZern A, O'Connell C, Roboz G, Busque L, Buckstein R, Amin H, Leber B, Shastri A, Oganesian A, Keer H, Azab M, Savona M. A plain language summary of the ASCERTAIN trial: oral decitabine and cedazuridine versus intravenous decitabine for MDS or CMML. Future Oncology 2025, 21: 929-941. PMID: 40051275, PMCID: PMC11938952, DOI: 10.1080/14796694.2025.2468578.Peer-Reviewed Original ResearchChronic myelomonocytic leukemiaAcute myeloid leukemiaIntravenous decitabineMyelodysplastic syndromeDEC-COral decitabineSide effectsRed blood cell transfusionPhase 3 clinical trialsMedian overall survivalTreatment-related deathsBlood cell transfusionDays of treatmentTransfusion independenceOverall survivalCell transfusionDecitabine groupMyelomonocytic leukemiaMyeloid leukemiaOral medicineDecitabineQuality of lifeReceiving treatmentTaking treatmentCedazuridineCharacterization of sulopenem antimicrobial activity using in vitro time-kill kinetics, synergy, post-antibiotic effect, and sub-inhibitory MIC effect methods against Escherichia coli and Klebsiella pneumoniae isolates
Maher J, Huband M, Lindley J, Rhomberg P, Aronin S, Puttagunta S, Castanheira M. Characterization of sulopenem antimicrobial activity using in vitro time-kill kinetics, synergy, post-antibiotic effect, and sub-inhibitory MIC effect methods against Escherichia coli and Klebsiella pneumoniae isolates. Microbiology Spectrum 2025, 13: e01898-24. PMID: 39907459, PMCID: PMC11878024, DOI: 10.1128/spectrum.01898-24.Peer-Reviewed Original ResearchConceptsUncomplicated urinary tract infectionsPost-antibiotic effectIntra-abdominal infectionsTreatment of urinary tractTime-kill testsMultidrug-resistant pathogensBaseline MICPenem antibioticUrinary tractPAE effectsClinical developmentBroth microdilution susceptibility testingPhase 3 clinical trialsKlebsiella pneumoniae isolatesOral dosing regimenUrinary tract infectionMicrodilution susceptibility testingPAE-SMETime-kill assayTime-kill kineticsStudy evaluated <i>inMg/500 mgAmoxicillin-clavulanateTract infectionsSusceptibility testing
2024
OVERTURE Phase 2 MRI Analysis Demonstrates Reduced Corpus Callosum Atrophy in Alzheimer's Disease
Da X, Hempel E, Hajos M, Kern R, Cimenser A. OVERTURE Phase 2 MRI Analysis Demonstrates Reduced Corpus Callosum Atrophy in Alzheimer's Disease. Alzheimer's & Dementia 2024, 20: e085446. PMCID: PMC11714734, DOI: 10.1002/alz.085446.Peer-Reviewed Original ResearchMagnetic resonance imagingMonth 3Midsagittal planeDiffusion tensor imagingPhase 3 clinical trialsRegional structural alterationsCorpus callosumT1w MRI imagesMonths of treatmentCommissural white matter tractsCorpus callosum atrophyWhite matter atrophyAlzheimer's diseaseSensory stimulationMonth 6CC atrophyClinical trialsSham groupWhite matter tractsGamma frequenciesVisual stimulationResonance imagingReal-World Safety of Esketamine Nasal Spray: A Comprehensive Analysis of Esketamine and Respiratory Depression
Chepke C, Shelton R, Sanacora G, Doherty T, Tsytsik P, Parker N. Real-World Safety of Esketamine Nasal Spray: A Comprehensive Analysis of Esketamine and Respiratory Depression. The International Journal Of Neuropsychopharmacology 2024, 27: pyae058. PMID: 39611487, PMCID: PMC11635826, DOI: 10.1093/ijnp/pyae058.Peer-Reviewed Original ResearchEsketamine nasal sprayRespiratory depressionAdverse eventsTreatment of treatment-resistant depressionStandardized Medical Dictionary for Regulatory ActivitiesPhase 3 clinical trialsTreatment-resistant depressionMitigation strategy programCentral respiratory depressionTreatment of depressive symptomsAcute suicidal ideationPostdose observation periodNo adverse eventsMedical Dictionary for Regulatory ActivitiesOral antidepressantNasal sprayCases meeting criteriaDepressive disorderPostmarketing settingPostapproval dataSuicidal ideationDepressive symptomsOxygen saturationPulse oximetryCase definitionSurvodutide for treatment of obesity: rationale and design of two randomized phase 3 clinical trials (SYNCHRONIZE™‐1 and ‐2)
Wharton S, le Roux C, Kosiborod M, Platz E, Brueckmann M, Jastreboff A, Hussain S, Pedersen S, Borowska L, Unseld A, Kloer I, Kaplan L, committees and investigators T. Survodutide for treatment of obesity: rationale and design of two randomized phase 3 clinical trials (SYNCHRONIZE™‐1 and ‐2). Obesity 2024, 33: 67-77. PMID: 39495965, PMCID: PMC11664303, DOI: 10.1002/oby.24184.Peer-Reviewed Original ResearchTreatment of obesityBaseline BMIRandomized phase 3 clinical trialOnce-weekly subcutaneous injectionsPhase 3 clinical trialsBaseline to weekDouble-blind trialObesity-related complicationsBody weight reductionAbsence of T2DSystolic blood pressureLiver fat contentType 2 diabetesMagnetic resonance imagingMeasures of glycemiaSecondary endpointsDosing flexibilityProportion of participantsLifestyle modificationInvestigated glucagonSubcutaneous injectionBlood pressureBody compositionResonance imagingObesityValidating new symptom emergence as a patient-centric outcome measure for PD clinical trials
Zou H, Stebbins G, Simuni T, Luo S, Cedarbaum J. Validating new symptom emergence as a patient-centric outcome measure for PD clinical trials. Parkinsonism & Related Disorders 2024, 128: 107118. PMID: 39353265, DOI: 10.1016/j.parkreldis.2024.107118.Peer-Reviewed Original ResearchPD clinical trialsClinical trialsEmergent symptomsPhase 3 clinical trialsPhase 3 studySlowing of disease progressionOutcome measuresParkinson's diseaseDe novo Parkinson's diseaseMDS-UPDRSItem-level dataFrequency of ESSymptomatic medicationsFrequent administrationMonths of observationEfficacy assessmentDisease progressionSymptom emergenceRating ScaleItem responsesIb and IIUrate elevationTrialsBeyond HMAs: Novel Targets and Therapeutic Approaches
Getz T, Bewersdorf J, Kewan T, Stempel J, Bidikian A, Shallis R, Stahl M, Zeidan A. Beyond HMAs: Novel Targets and Therapeutic Approaches. Seminars In Hematology 2024, 61: 358-369. PMID: 39389839, DOI: 10.1053/j.seminhematol.2024.08.001.Peer-Reviewed Original ResearchAcute myeloid leukemiaDelays progression to acute myeloid leukemiaHeterogeneous group of clonal hematopoietic disordersGroup of clonal hematopoietic disordersMolecular International Prognostic Scoring SystemRandomized phase 3 clinical trialProgression to acute myeloid leukemiaInternational Prognostic Scoring SystemLower-risk MDS patientsRisk stratification of patientsPhase 3 clinical trialsCombination of azacitidineHypomethylating agent combinationsCurrent treatment landscapeFirst line therapyPrognostic scoring systemBiomarker-directed therapiesClonal hematopoietic disordersLack of therapeutic agentsStratification of patientsEarly phase trialsErythropoiesis stimulating agentsTreated with therapiesVariable clinical featuresStandard of care706 - Real-world patient experience of upadacitinib-treated adults with atopic dermatitis: results from the SCALE-UP study
Silverberg J, Calimlim B, Fuxench Z, Altman K, Bensimon A, Liu J, Bunick C. 706 - Real-world patient experience of upadacitinib-treated adults with atopic dermatitis: results from the SCALE-UP study. British Journal Of Dermatology 2024, 191: ljae266.080. DOI: 10.1093/bjd/ljae266.080.Peer-Reviewed Original ResearchPatient support programAtopic dermatitisItch improvementSkin clearanceOral Janus kinase inhibitorPatient experiencePhase 3 clinical trialsTreating moderate-to-severe ADEffect of upadacitinibMultiple phase 3 clinical trialsModerate-to-severe ADJanus kinase inhibitorsClinical trial resultsPatient-reported experiencesDecreased quality of lifeEvaluate patient experiencePatient-reported outcomesTopical corticosteroidsPatient ageCross-sectional analysisQuality of lifeUpadacitinib dosesKinase inhibitorsUpadacitinibTreatment durationFactor XI: structure, function and therapeutic inhibition
Ali A, Becker R. Factor XI: structure, function and therapeutic inhibition. Journal Of Thrombosis And Thrombolysis 2024, 57: 1315-1328. PMID: 38622277, PMCID: PMC11645426, DOI: 10.1007/s11239-024-02972-5.Peer-Reviewed Original ResearchFactor XI inhibitorsVitamin K antagonistsEnd-stage renal diseaseFactor XITraditional anticoagulantsSafety profileInherent risk of bleedingPhase 3 clinical trialsPhase 2 studyMultiple coagulation factorsRisk of bleedingFactor XI deficiencyIntrinsic pathway of coagulationPathway of coagulationFXI inhibitorsK antagonistsOral anticoagulantsAnticoagulant therapyTherapeutic anticoagulationVenous thromboembolismPreclinical studiesAtrial fibrillationClinical endpointsRenal diseaseClinical trialsThree- and Seven-month Prostate-specific Antigen Levels as Prognostic Markers for Overall Survival in Metastatic Hormone-sensitive Prostate Cancer: Results from SWOG S1216, a Phase 3 Randomized Trial of Androgen Deprivation Plus Orteronel or Bicalutamide
Parikh M, Tangen C, Hussain M, Gupta S, Callis S, Jo Y, Harzstark A, Paller C, George S, Zibelman M, Cheng H, Maughan B, Zhang J, Pachynski R, Bryce A, Lin D, Quinn D, Lerner S, Thompson I, Dorff T, Lara P, Agarwal N. Three- and Seven-month Prostate-specific Antigen Levels as Prognostic Markers for Overall Survival in Metastatic Hormone-sensitive Prostate Cancer: Results from SWOG S1216, a Phase 3 Randomized Trial of Androgen Deprivation Plus Orteronel or Bicalutamide. European Urology Oncology 2024, 7: 1097-1104. PMID: 38523017, PMCID: PMC12037053, DOI: 10.1016/j.euo.2024.03.001.Peer-Reviewed Original ResearchMetastatic hormone-sensitive prostate cancerHormone-sensitive prostate cancerProstate-specific antigenAndrogen deprivation therapyProstate-specific antigen levelOverall survivalProstate cancerDeprivation therapyComplete responseAntigen levelsNo responseTreatment of metastatic hormone-sensitive prostate cancerLow prostate-specific antigen levelsResponse to androgen deprivation therapyAssociated with improved OSPSA responsePhase 3 randomized trialPhase 3 clinical trialsEarly identification of patientsAssociated with OSHigher risk of deathIdentification of patientsResponse to treatmentRisk of deathOS associationMeaningful Change Thresholds for the Psoriasis Symptoms and Signs Diary
Papp K, Gordon K, Strober B, Zhuo J, Becker B, Zhong Y, Beaumont J, Pham T, Kisa R, Napoli A, Banerjee S, Armstrong A. Meaningful Change Thresholds for the Psoriasis Symptoms and Signs Diary. JAMA Dermatology 2024, 160: 204-209. PMID: 38117487, PMCID: PMC10733845, DOI: 10.1001/jamadermatol.2023.5058.Peer-Reviewed Original ResearchSevere plaque psoriasisPatient Global ImpressionClinical trialsPsoriasis symptomsScore changeSigns DiaryPlaque psoriasisGlobal ImpressionWeek 16MAIN OUTCOMEPatient-reported end pointsPlacebo-controlled phase 3 clinical trialPhase 3 clinical trialsPatient-reported assessmentsPhase 3 programMeaningful change thresholdCategory improvementDisease burdenMeaningful score changesSelective TYK2 inhibitorsPatient changeBaseline scoresPsoriasisPatientsEnd pointBimekizumab Efficacy Through 3 Years in Patients with Moderate to Severe Plaque Psoriasis: Long-term Pooled Analysis from BE BRIGHT
Lebwohl M, Strober B, Foley P, Langley R, Tada Y, Hampton P, Davis L, Wiegratz S, Hoepken B, Lambert J, Kokolakis G. Bimekizumab Efficacy Through 3 Years in Patients with Moderate to Severe Plaque Psoriasis: Long-term Pooled Analysis from BE BRIGHT. SKIN The Journal Of Cutaneous Medicine 2024, 8: s307. DOI: 10.25251/skin.8.supp.307.Peer-Reviewed Original ResearchOpen-label extensionDermatology Life Quality IndexSevere plaque psoriasisPhase 3 trialPlaque psoriasisPooled analysisDermatology Life Quality Index 0/1Discontinued treatment due to lackModerate to severe plaque psoriasisOLE weekPooled analysis of patientsTreatment-related adverse eventsPhase 3 clinical trialsEfficacy of bimekizumabPlaque psoriasis patientsTreatment due to lackAnalysis of patientsLong-term treatment efficacyProportion of patientsNext scheduled visitNon-responder imputationLoss of responseQuality of life responsesLife Quality IndexBE VIVIDBatoclimab as induction and maintenance therapy in patients with myasthenia gravis: rationale and study design of a phase 3 clinical trial
Benatar M, Wiendl H, Nowak R, Zheng Y, Macias W. Batoclimab as induction and maintenance therapy in patients with myasthenia gravis: rationale and study design of a phase 3 clinical trial. BMJ Neurology Open 2024, 6: e000536. PMID: 38268752, PMCID: PMC10806862, DOI: 10.1136/bmjno-2023-000536.Peer-Reviewed Original ResearchMG-ADLMyasthenia gravisInstitutional Review Board/Independent Ethics CommitteePhase 3 clinical trialsPlacebo-treated patientsPlacebo-controlled studyBaseline to weekHuman monoclonal antibodyLong-term extensionMyasthenia Gravis ActivitiesWritten Informed ConsentMaintenance periodInduction therapyMaintenance therapyDeclaration of HelsinkiPrimary endpointMaintenance treatmentQuadruple-blindSubcutaneous injectionPatientsPlaceboMonoclonal antibodiesMyastheniaInformed consentQ2WThe greatest Dengue epidemic in Brazil: Surveillance, Prevention, and Control
Gurgel-Gonçalves R, de Oliveira W, Croda J. The greatest Dengue epidemic in Brazil: Surveillance, Prevention, and Control. Revista Da Sociedade Brasileira De Medicina Tropical 2024, 57: e00203-2024. PMID: 39319953, PMCID: PMC11415067, DOI: 10.1590/0037-8682-0113-2024.Peer-Reviewed Original ResearchConceptsHealth education initiativesPhase 3 clinical trialsPublic health systemMinistry of HealthIndoor residual sprayingSterile insect techniqueHealth servicesHealth systemTakeda vaccineCommunity mobilizationMultisectoral approachDengue epidemicsDengue casesEducational initiativesDengue mortalityResidual sprayingMosquito controlHome cleaningInsect techniqueAedes aegyptiPotential mosquito breeding sitesHealthVaccination coverageDengue virusVaccine
2023
Cost-Effectiveness of Long-Term Prophylaxis with Plasma-Derived Vs. Recombinant Von Willebrand Factor in Severe Von Willebrand Disease
Waldron C, Ito S, Wang D, Allen C, Viswanathan G, Bona R, Cuker A, Goshua G. Cost-Effectiveness of Long-Term Prophylaxis with Plasma-Derived Vs. Recombinant Von Willebrand Factor in Severe Von Willebrand Disease. Blood 2023, 142: 3698. DOI: 10.1182/blood-2023-188018.Peer-Reviewed Original ResearchSevere von Willebrand diseaseLong-term prophylaxisIncremental net monetary benefitVon Willebrand diseasePlasma-derived von Willebrand factorIncremental cost-effectiveness ratioNet monetary benefitVon Willebrand factorProbabilistic sensitivity analysesDeterministic sensitivity analysesSevere haemophiliaWillebrand factorRecombinant von Willebrand factorWillebrand diseasePhase 3 clinical trialsHealth resource utilizationFactor replacement therapyFrequency of infusionsSubset of patientsCost of prophylaxisType 3 von Willebrand diseaseFactor replacement productsSimilar significant improvementsSevere phenotypeNon-significant reductionPilot study suggests DNA methylation of the glucocorticoid receptor gene (NR3C1) is associated with MDMA-assisted therapy treatment response for severe PTSD
Lewis C, Tafur J, Spencer S, Green J, Harrison C, Kelmendi B, Rabin D, Yehuda R, Yazar-Klosinski B, Cahn B. Pilot study suggests DNA methylation of the glucocorticoid receptor gene (NR3C1) is associated with MDMA-assisted therapy treatment response for severe PTSD. Frontiers In Psychiatry 2023, 14: 959590. PMID: 36815187, PMCID: PMC9939628, DOI: 10.3389/fpsyt.2023.959590.Peer-Reviewed Original ResearchPost-traumatic stress disorderSevere post-traumatic stress disorderTreatment responseClinical trialsRecent phase 3 clinical trialsPhase 3 clinical trialsClinician-Administered PTSD ScaleParent clinical trialKey HPA axis genesHPA axis genesPTSD symptom improvementGlucocorticoid receptor geneFalse discovery rate correctionAdrenal genesPlacebo groupSymptom improvementLarge cohortMethylation changesEpigenetic changesSymptom reductionPTSD ScaleTherapyHPA genesStress disorderPilot study
2022
Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors
Pipe S, Hermans C, Chitlur M, Carcao M, Castaman G, Davis J, Ducore J, Dunn A, Escobar M, Journeycake J, Khan O, Mahlangu J, Meeks S, Mitha I, Négrier C, Nowak‐Göttl U, Recht M, Chrisentery‐Singleton T, Stasyshyn O, Vilchevska K, Martinez L, Wang M, Windyga J, Young G, Alexander W, Bonzo D, Macie C, Mitchell I, Sauty E, Wilkinson T, Shapiro A. Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors. Haemophilia 2022, 28: 548-556. PMID: 35475308, PMCID: PMC9542908, DOI: 10.1111/hae.14563.Peer-Reviewed Original ResearchConceptsInitial dosing regimensControl of bleedingYears of ageEptacog betaBleeding episodesHemophilia ATreatment-related adverse eventsPhase 3 clinical trialsAlternative haemostatic agentModerate bleeding episodesTreat bleeding episodesTreatment of bleedingTreatment success criteriaSuccess proportionRandomised crossover designDosing regimensBeta infusionPaediatric subjectsAdverse eventsBlood productsHaemostatic agentsBleedingEffective treatmentAllergic reactionsNeutralising antibodies
2021
The safety of activated eptacog beta in the management of bleeding episodes and perioperative haemostasis in adult and paediatric haemophilia patients with inhibitors
Escobar M, Castaman G, Boix S, Callaghan M, de Moerloose P, Ducore J, Hermans C, Journeycake J, Leissinger C, Luck J, Mahlangu J, Miesbach W, Mitha I, Négrier C, Quon D, Recht M, Schved J, Shapiro A, Sidonio R, Srivastava A, Stasyshyn O, Vilchevska K, Wang M, Young G, Alexander W, Al‐Sabbagh A, Bonzo D, Macie C, Wilkinson T, Kessler C. The safety of activated eptacog beta in the management of bleeding episodes and perioperative haemostasis in adult and paediatric haemophilia patients with inhibitors. Haemophilia 2021, 27: 921-931. PMID: 34636112, PMCID: PMC9292935, DOI: 10.1111/hae.14419.Peer-Reviewed Original ResearchConceptsEptacog betaTreatment of bleedingB patientsBleeding episodesHemophilia ASafety profileHemophilia patientsProspective phase 3 clinical trialsManagement of bleeding episodesPhase 3 clinical trialsTreat bleeding episodesPhase 3 trialControl of bleedingExcellent safety profilePaediatric haemophilia patientsPerioperative haemostasisPreoperative infusionClinical responseRandomized crossover designPerioperative useThrombotic eventsPost-operativelyData monitoring committeeFDA approvalPerioperative careAssociation of BNT162b2 mRNA and mRNA-1273 Vaccines With COVID-19 Infection and Hospitalization Among Patients With Cirrhosis
John BV, Deng Y, Scheinberg A, Mahmud N, Taddei TH, Kaplan D, Labrada M, Baracco G, Dahman B. Association of BNT162b2 mRNA and mRNA-1273 Vaccines With COVID-19 Infection and Hospitalization Among Patients With Cirrhosis. JAMA Internal Medicine 2021, 181: 1306-1314. PMID: 34254978, PMCID: PMC8278308, DOI: 10.1001/jamainternmed.2021.4325.Peer-Reviewed Original ResearchConceptsCOVID-19 infectionCOVID-19-related hospitalizationBNT162b2 mRNAFirst doseCohort studySecond doseMRNA vaccinesClinical trialsControl groupPropensity-matched control groupPhase 3 clinical trialsCOVID-19 mRNA vaccinesAssociation of vaccinationRetrospective cohort studyUS veteran populationAssociation of receiptVeterans Health AdministrationCoronavirus disease 2019COVID-19 hospitalizationMRNA vaccine administrationCOVID-19Vaccine recipientsImmune dysregulationModerna mRNAMedian age
This site is protected by hCaptcha and its Privacy Policy and Terms of Service apply