2025
Generics, Biosimilars and Follow‐On Non‐Biologic Complex Drugs for Multiple Sclerosis: A Narrative Review of the Regulatory and Clinical Implications for European Neurologists
Berger T, Zeitlinger M, Popescu V, Magyari M, Airas L, Alkhawajah M, Pugliatti M, Zakaria M, Pozzilli C, Drulovic J, Van Wijmeersch B, Vermersch P, Oreja‐Guevara C. Generics, Biosimilars and Follow‐On Non‐Biologic Complex Drugs for Multiple Sclerosis: A Narrative Review of the Regulatory and Clinical Implications for European Neurologists. European Journal Of Neurology 2025, 32: e70140. PMID: 40231751, PMCID: PMC11998027, DOI: 10.1111/ene.70140.Peer-Reviewed Original ResearchConceptsDisease-modifying treatmentsNon-biological complex drugsImprove patient accessLiterature searchMultiple sclerosisMS careHealthcare systemPatient accessComplex drugsPubMed literature searchSocioeconomic burdenNarrative reviewTolerability profileGlatiramer acetateClinical efficacyClinical studiesEarly onsetEuropean neurologistsClinical implicationsProgressive natureDrugSclerosisOriginal drugPeopleCareTranslational genomics of osteoarthritis in 1,962,069 individuals
Hatzikotoulas K, Southam L, Stefansdottir L, Boer C, McDonald M, Pett J, Park Y, Tuerlings M, Mulders R, Barysenka A, Arruda A, Tragante V, Rocco A, Bittner N, Chen S, Horn S, Srinivasasainagendra V, To K, Katsoula G, Kreitmaier P, Tenghe A, Gilly A, Arbeeva L, Chen L, de Pins A, Dochtermann D, Henkel C, Höijer J, Ito S, Lind P, Lukusa-Sawalena B, Minn A, Mola-Caminal M, Narita A, Nguyen C, Reimann E, Silberstein M, Skogholt A, Tiwari H, Yau M, Yue M, Zhao W, Zhou J, Alexiadis G, Banasik K, Brunak S, Campbell A, Cheung J, Dowsett J, Faquih T, Faul J, Fei L, Fenstad A, Funayama T, Gabrielsen M, Gocho C, Gromov K, Hansen T, Hudjashov G, Ingvarsson T, Johnson J, Jonsson H, Kakehi S, Karjalainen J, Kasbohm E, Lemmelä S, Lin K, Liu X, Loef M, Mangino M, McCartney D, Millwood I, Richman J, Roberts M, Ryan K, Samartzis D, Shivakumar M, Skou S, Sugimoto S, Suzuki K, Takuwa H, Teder-Laving M, Thomas L, Tomizuka K, Turman C, Weiss S, Wu T, Zengini E, Zhang Y, Ferreira M, Babis G, Baras A, Barker T, Carey D, Cheah K, Chen Z, Cheung J, Daly M, de Mutsert R, Eaton C, Erikstrup C, Furnes O, Golightly Y, Gudbjartsson D, Hailer N, Hayward C, Hochberg M, Homuth G, Huckins L, Hveem K, Ikegawa S, Ishijima M, Isomura M, Jones M, Kang J, Kardia S, Kloppenburg M, Kraft P, Kumahashi N, Kuwata S, Lee M, Lee P, Lerner R, Li L, Lietman S, Lotta L, Lupton M, Mägi R, Martin N, McAlindon T, Medland S, Michaëlsson K, Mitchell B, Mook-Kanamori D, Morris A, Nabika T, Nagami F, Nelson A, Ostrowski S, Palotie A, Pedersen O, Rosendaal F, Sakurai-Yageta M, Schmidt C, Sham P, Singh J, Smelser D, Smith J, Song Y, Sørensen E, Tamiya G, Tamura Y, Terao C, Thorleifsson G, Troelsen A, Tsezou A, Uchio Y, Uitterlinden A, Ullum H, Valdes A, van Heel D, Walters R, Weir D, Wilkinson J, Winsvold B, Yamamoto M, Zwart J, Stefansson K, Meulenbelt I, Teichmann S, van Meurs J, Styrkarsdottir U, Zeggini E. Translational genomics of osteoarthritis in 1,962,069 individuals. Nature 2025, 641: 1217-1224. PMID: 40205036, PMCID: PMC12119359, DOI: 10.1038/s41586-025-08771-z.Peer-Reviewed Original ResearchConceptsEffector genesGenome-wide association study meta-analysesTargets of approved drugsVariant associationsTranslational genomicsEpigenomic profilingStudy meta-analysesCircadian clockBiological processesLines of evidenceConditions associated with disabilityRepurposing opportunitiesSignal enrichmentGenesEffectorPathwayIndependent associationsMeta-analysesEffect sizeAccelerated translationEpigenomeTranscriptomeProteomicsDisease-modifying treatmentsOsteoarthritisMetabolic Phenotype of Stage 1 and Stage 2 Type 1 Diabetes Using Modeling of β Cell Function
Galderisi A, Bonet J, Ismail H, Moran A, Fiorina P, Bosi E, Petrelli A. Metabolic Phenotype of Stage 1 and Stage 2 Type 1 Diabetes Using Modeling of β Cell Function. The Journal Of Clinical Endocrinology & Metabolism 2025, dgaf086. PMID: 39992257, DOI: 10.1210/clinem/dgaf086.Peer-Reviewed Original ResearchOral glucose tolerance testMetabolic phenotypeOral minimal modelInsulin secretionResponse to disease-modifying treatmentsOdds of disease progressionPhenotype of individualsPrevention trialsPreclinical type 1 diabetesIndependent of stageMeasures of beta-cell functionStage 1Glucose tolerance testAssociated with reduced oddsBeta-cell functionMeasures of insulin secretionType 1 diabetesOGTT-derived measuresIncreased insulin clearanceT1D prevention trialsIslet autoimmunityDisease-modifying treatmentsTolerance testDisease progressionProgression predictorsAn international perspective on the future of systemic sclerosis research
Abraham D, Black C, Denton C, Distler J, Domsic R, Feghali-Bostwick C, Gourh P, Hinchcliff M, Kolling F, Kuwana M, Lafyatis R, Landegren U, Mahoney J, Martin J, Matucci-Cerinic M, McMahan Z, Mora A, Mouthon L, Rabinovitch M, Rojas M, Rubin K, Trojanowska M, Varga J, Whitfield M, Gabrielli A, Krieg T. An international perspective on the future of systemic sclerosis research. Nature Reviews Rheumatology 2025, 21: 174-187. PMID: 39953141, DOI: 10.1038/s41584-024-01217-2.Peer-Reviewed Original ResearchConceptsSystemic sclerosisSystemic sclerosis researchHistory of systemic sclerosisInnovative clinical trial designsSystemic autoimmune diseaseEarly pathogenetic eventClinical trial designCollaborative global effortAutoimmune phenomenaImprove patient outcomesDisease activityMicrovascular dysfunctionClinical historyEmergency therapyAutoimmune diseasesDisease-modifying treatmentsTissue fibrosisSingle-cell RNA sequencingClinical heterogeneityEffective disease-modifying treatmentsPersonalized therapyAnimal modelsComplex pathogenesisEffective treatmentNext-generation sequencingAnti-Amyloid Therapies for Alzheimer’s Disease and Amyloid-Related Imaging Abnormalities: Implications for the Emergency Medicine Clinician
Rech M, Carpenter C, Aggarwal N, Hwang U. Anti-Amyloid Therapies for Alzheimer’s Disease and Amyloid-Related Imaging Abnormalities: Implications for the Emergency Medicine Clinician. Annals Of Emergency Medicine 2025, 85: 526-536. PMID: 39818674, DOI: 10.1016/j.annemergmed.2024.12.002.Peer-Reviewed Original ResearchEmergency medicine cliniciansAnti-amyloid therapiesCases of dementiaEmergency department visitsAlzheimer's diseaseDepartment visitsIschemic stroke patientsStroke patientsIncreased riskAlzheimer's disease progressionCliniciansRisk of adverse effectsAnti-amyloidDisease-modifying treatmentsAmyloid-relatedNeurodegenerative disordersImaging abnormalitiesAlzheimerAdverse effectsEmergencyDementiaAmyloid-related imaging abnormalities
2024
Real-world infection risk in multiple sclerosis patients on long-term immunomodulatory treatments
Lambert C, Hussain T, Peters J, Longbrake E. Real-world infection risk in multiple sclerosis patients on long-term immunomodulatory treatments. Multiple Sclerosis And Related Disorders 2024, 94: 106236. PMID: 39755026, PMCID: PMC11869388, DOI: 10.1016/j.msard.2024.106236.Peer-Reviewed Original ResearchConceptsCharlson Comorbidity IndexAnti-CD20 medicationsDisease-modifying treatmentsDMT useS1P modulatorsImmunomodulatory treatmentImmune cellsSevere infectionsMultiple sclerosisLong-term immunomodulatory treatmentReal-life practice patternsTreatment durationInfection rateSelection of patientsClinical selection of patientsDeplete immune cellsObservational cohort studyBody mass indexSevere infection rateAnalyzed medical recordsIncreased infection riskInfection riskDisease modifying therapiesMultiple sclerosis patientsImmune subsetsEarly Disease-Modifying Treatments for Presymptomatic Multiple Sclerosis
Zeydan B, Azevedo C, Makhani N, Cohen M, Tutuncu M, Thouvenot E, Siva A, Okuda D, Kantarci O, Lebrun-Frenay C. Early Disease-Modifying Treatments for Presymptomatic Multiple Sclerosis. CNS Drugs 2024, 38: 973-983. PMID: 39285136, PMCID: PMC11560559, DOI: 10.1007/s40263-024-01117-9.Peer-Reviewed Original ResearchRadiologically isolated syndromeRisk factorsLaboratory biomarkersMultiple sclerosisDisease-modifying treatmentsSpinal cordGadolinium-enhancing lesionsEfficacy of disease-modifying treatmentsRandomized clinical trialsIncrease diagnostic accuracyLack of clinical guidelinesSymptomatic MSNeurofilament-light chainCSF abnormalitiesPresymptomatic individualsAdverse eventsMale sexMS criteriaClinical eventsDiagnostic accuracyClinical trialsDisease outcomeClinical guidelinesYounger ageLesionsIon channels in osteoarthritis: emerging roles and potential targets
Zhou R, Fu W, Vasylyev D, Waxman S, Liu C. Ion channels in osteoarthritis: emerging roles and potential targets. Nature Reviews Rheumatology 2024, 20: 545-564. PMID: 39122910, DOI: 10.1038/s41584-024-01146-0.Peer-Reviewed Original ResearchIon channelsVoltage-dependent calcium channelsAcid-sensing ion channelsTransient receptor potential channelsVoltage-gated sodium channelsIon channel modulatorsFunction of ion channelsPotential clinical applicationsCalcium channelsPreclinical studiesClinical impactSymptomatic reliefPotassium channelsChloride channelsDisease-modifying treatmentsClinical trialsSodium channelsBone hyperplasiaChannel modulationIon channel biologySynovial inflammationClinical applicationPiezo channelsModel of OAPotential targetManagement of postinfectious inflammatory arthritis
Baker H, Amaral J, Schoen R. Management of postinfectious inflammatory arthritis. Current Opinion In Rheumatology 2024, 36: 155-162. PMID: 38411201, DOI: 10.1097/bor.0000000000001009.Peer-Reviewed Original ResearchConceptsTreatment of acute infectionsAcute infectionInflammatory arthritisBenefits of antimicrobial therapyTreatment of infectionsTargeted synthetic DMARDsSpectrum of diseasesRefractory arthritisAntimicrobial therapySynthetic DMARDsFraction of casesTreatment optionsConventional therapyDisease-modifying treatmentsReactive arthritisInflammatory arthritidesInflammatory processPrompt identificationSelf-limitingChronic arthritisArthritisInfectionTreatmentTherapyArthralgiaA review of Bruton’s tyrosine kinase inhibitors in multiple sclerosis
Airas L, Bermel R, Chitnis T, Hartung H, Nakahara J, Stuve O, Williams M, Kieseier B, Wiendl H. A review of Bruton’s tyrosine kinase inhibitors in multiple sclerosis. Therapeutic Advances In Neurological Disorders 2024, 17: 17562864241233041. PMID: 38638671, PMCID: PMC11025433, DOI: 10.1177/17562864241233041.Peer-Reviewed Original ResearchBruton tyrosine kinase inhibitorChronic spontaneous urticariaFirst-generation BTK inhibitorSystemic lupus erythematosusBruton's tyrosine kinaseTyrosine kinase inhibitorsMS disease-modifying treatmentsBTK inhibitorsDisease-modifying treatmentsSafety profileB cellsMultiple sclerosisChronic useRheumatoid arthritisSecond-generation BTK inhibitorsB cell depletion therapyTreatment of B-cell malignanciesB-cell modulationDiverse autoimmune conditionsPhase II trialGadolinium-enhancing lesionsB-cell malignanciesMagnetic resonance imaging scansLong-term administrationFemale reproductive health
2023
Calcium signaling in chemotherapy-induced neuropathy
Fonseca M, Marazzi-Diniz P, Leite M, Ehrlich B. Calcium signaling in chemotherapy-induced neuropathy. Cell Calcium 2023, 113: 102762. PMID: 37244172, DOI: 10.1016/j.ceca.2023.102762.Peer-Reviewed Original ResearchConceptsChemotherapy-induced peripheral neuropathyNeuronal calcium sensor-1Chemotherapy-induced cognitive impairmentMultiple chemotherapy regimensChemotherapy-induced neuropathyDisease-modifying treatmentsQuality of lifeChemotherapy regimensChemotherapy dosePeripheral neuropathyPathophysiological cascadePatient outcomesOptimal chemotherapySide effectsCognitive impairmentChemotherapeutic agentsTarget toxicityCancer cellsSensitive CaCalcium signalingOff-target effectsNeuropathyTaxanesTreatmentMolecular mechanisms
2022
Targeted Therapies in the Treatment of Uterine Serous Carcinoma
Tymon-Rosario J, Gorman M, Santin A. Targeted Therapies in the Treatment of Uterine Serous Carcinoma. Current Treatment Options In Oncology 2022, 23: 1804-1817. PMID: 36447064, DOI: 10.1007/s11864-022-01030-7.Peer-Reviewed Original ResearchConceptsUterine serous carcinomaRecurrent diseaseSerous carcinomaTreatment of USCEffective disease-modifying treatmentMolecular classificationAppropriate surgical stagingAddition of trastuzumabCombination of immunotherapySystemic adjuvant treatmentDisease-modifying treatmentsEnrollment of patientsHER2-positive tumorsTyrosine kinase inhibitorsUnmet medical needPersonalized cancer careAntibody-drug conjugatesOpinion statementDespiteSurgical cytoreductionAdjuvant treatmentSurgical stagingDismal prognosisSurvival outcomesTherapeutic optionsCancer careTrial of Cinpanemab in Early Parkinson’s Disease
Lang A, Siderowf A, Macklin E, Poewe W, Brooks D, Fernandez H, Rascol O, Giladi N, Stocchi F, Tanner C, Postuma R, Simon D, Tolosa E, Mollenhauer B, Cedarbaum J, Fraser K, Xiao J, Evans K, Graham D, Sapir I, Inra J, Hutchison R, Yang M, Fox T, Budd Haeberlein S, Dam T. Trial of Cinpanemab in Early Parkinson’s Disease. New England Journal Of Medicine 2022, 387: 408-420. PMID: 35921450, DOI: 10.1056/nejmoa2203395.Peer-Reviewed Original ResearchConceptsEarly Parkinson's diseasePrimary end pointSecondary end pointsWeek 52Parkinson's diseaseEnd pointControl groupUnified Parkinson's Disease Rating Scale (UPDRS) total scoreDisease pathogenesisHuman-derived monoclonal antibodiesΑ-synucleinCommon adverse eventsPhase 2 trialDisease-modifying treatmentsMDS-UPDRS scoresLack of efficacyDAT SPECT imagingAdjusted mean differenceMovement Disorder SocietyParkinson's disease pathogenesisScale total scoreAdverse eventsIntravenous infusionDAT-SPECTDisease progression
2021
Engaging Caregivers and Providers of Children With Sickle Cell Anemia in Shared Decision Making for Hydroxyurea: Protocol for a Multicenter Randomized Controlled Trial
Hood AM, Strong H, Nwankwo C, Johnson Y, Peugh J, Mara CA, Shook LM, Brinkman WB, Real FJ, Klein MD, Hackworth R, Badawy SM, Thompson AA, Raphael JL, Yates AM, Smith-Whitley K, King AA, Calhoun C, Creary SE, Piccone CM, Hildenbrand AK, Reader SK, Neumayr L, Meier ER, Sobota AE, Rana S, Britto M, Saving KL, Treadwell M, Quinn CT, Ware RE, Crosby LE. Engaging Caregivers and Providers of Children With Sickle Cell Anemia in Shared Decision Making for Hydroxyurea: Protocol for a Multicenter Randomized Controlled Trial. JMIR Research Protocols 2021, 10: e27650. PMID: 34018965, PMCID: PMC8178738, DOI: 10.2196/27650.Peer-Reviewed Original ResearchSickle cell anemiaINTERNATIONAL REGISTERED REPORT IDENTIFIERHospital Medical CenterCell anemiaCincinnati Children's Hospital Medical CenterOnly disease-modifying treatmentHigher health care utilizationPotential long-term side effectsChildren's Hospital Medical CenterLong-term side effectsDisease-modifying treatmentsCaregivers of patientsHealth care utilizationEvidence-based guidelinesSerious medical complicationsResultant health outcomesSickle cell diseaseLife-threatening diseaseQuality of careHigh-quality careGenetic blood disorderProviders of childrenEarly morbidityControlled TrialsMedical complicationsLongitudinal Changes in Neuromelanin MRI Signal in Parkinson's Disease: A Progression Marker
Gaurav R, Yahia‐Cherif L, Pyatigorskaya N, Mangone G, Biondetti E, Valabrègue R, Ewenczyk C, Hutchison R, Cedarbaum J, Corvol J, Vidailhet M, Lehéricy S. Longitudinal Changes in Neuromelanin MRI Signal in Parkinson's Disease: A Progression Marker. Movement Disorders 2021, 36: 1592-1602. PMID: 33751655, PMCID: PMC8359265, DOI: 10.1002/mds.28531.Peer-Reviewed Original ResearchConceptsTotal intracranial volumePD patientsHealthy volunteersParkinson's diseaseIntracranial volumeObservational case-control studySubstantia nigra pars compactaDirect noninvasive assessmentDisease-modifying treatmentsEarly PD patientsFemale PD patientsTherapeutic drug trialsCase-control studyNeuromelanin-sensitive MRI techniquesNeuromelanin-sensitive imagingMale patientsFemale patientsPars compactaCohort IICohort IDrug trialsPatientsImaging biomarkersNoninvasive assessmentDisease severity
2020
Treatment Considerations in the Radiologically Isolated Syndrome
Makhani N. Treatment Considerations in the Radiologically Isolated Syndrome. Current Treatment Options In Neurology 2020, 22: 3. PMID: 32009206, DOI: 10.1007/s11940-020-0608-8.Peer-Reviewed Original ResearchClinical intervention trialsMultiple sclerosisIntervention trialsClinical neurological symptomsFirst clinical eventDisease-modifying therapiesDisease-modifying treatmentsIsolated SyndromeNeurological eventsNeurological symptomsClinical eventsClinical trialsRecent FindingsIndividualsTreatment considerationsTherapeutic interventionsAccurate diagnosisBeneficial effectsTrialsSclerosisSuch interventionsSyndromeSubsequent developmentDiagnosisInterventionTherapy
2018
Assessing Synaptic Density in Alzheimer Disease With Synaptic Vesicle Glycoprotein 2A Positron Emission Tomographic Imaging
Chen MK, Mecca AP, Naganawa M, Finnema SJ, Toyonaga T, Lin SF, Najafzadeh S, Ropchan J, Lu Y, McDonald JW, Michalak HR, Nabulsi NB, Arnsten AFT, Huang Y, Carson RE, van Dyck CH. Assessing Synaptic Density in Alzheimer Disease With Synaptic Vesicle Glycoprotein 2A Positron Emission Tomographic Imaging. JAMA Neurology 2018, 75: 1215-1224. PMID: 30014145, PMCID: PMC6233853, DOI: 10.1001/jamaneurol.2018.1836.Peer-Reviewed Original ResearchConceptsPositron emission tomographic imagingSynaptic vesicle glycoprotein 2ASynaptic densityAlzheimer's diseaseEmission tomographic imagingHigh-resolution PET scanningPET scanningCognitive impairmentDisease-modifying therapiesDisease-modifying treatmentsNormal participantsCross-sectional studyPittsburgh compound BMajor structural correlateAmnestic mild cognitive impairmentMagnetic resonance imagingMild cognitive impairmentJ PET imagingRestoration of synapsesSpecific bindingNeurologic evaluationSynaptic lossDisease stagePostmortem studiesOutcome measuresTargeted Therapies for Parkinson's Disease: From Genetics to the Clinic
Sardi SP, Cedarbaum JM, Brundin P. Targeted Therapies for Parkinson's Disease: From Genetics to the Clinic. Movement Disorders 2018, 33: 684-696. PMID: 29704272, PMCID: PMC6282975, DOI: 10.1002/mds.27414.Peer-Reviewed Original ResearchConceptsParkinson's diseaseGreat unmet medical needDisease-modifying treatmentsNew therapeutic approachesUnmet medical needClinical stageClinical trialsRelentless progressionTherapeutic approachesPotential therapyClinical developmentTherapeutic paradigmMedical needDiseaseGenetic variantsPD geneticsNew arsenalTreatmentGenetic discoveriesKey outstanding questionsTherapySymptomsPathologyProgressionTrials
2017
Anti-Amyloid-β Monoclonal Antibodies for Alzheimer’s Disease: Pitfalls and Promise
van Dyck CH. Anti-Amyloid-β Monoclonal Antibodies for Alzheimer’s Disease: Pitfalls and Promise. Biological Psychiatry 2017, 83: 311-319. PMID: 28967385, PMCID: PMC5767539, DOI: 10.1016/j.biopsych.2017.08.010.Peer-Reviewed Original ResearchConceptsAlzheimer's diseaseAmyloid-related imaging abnormalitiesPutative disease-modifying treatmentsMonoclonal antibodiesOngoing prevention trialsDisease-modifying treatmentsPresymptomatic Alzheimer's diseaseImaging abnormalitiesPrevention trialsClinical efficacyPassive immunizationClinical trialsTherapeutic approachesDisease processPreclinical stageAβ-MAbNew trialsDiseaseAdditional studiesTrialsBest treatmentMAbsTreatmentAntibodiesConformation of Aβ
2016
The need for new approaches in CNS drug discovery: Why drugs have failed, and what can be done to improve outcomes
Gribkoff VK, Kaczmarek LK. The need for new approaches in CNS drug discovery: Why drugs have failed, and what can be done to improve outcomes. Neuropharmacology 2016, 120: 11-19. PMID: 26979921, PMCID: PMC5820030, DOI: 10.1016/j.neuropharm.2016.03.021.Peer-Reviewed Original ResearchConceptsCNS drug discoveryClinical failure rateDisease-modifying treatmentsEffects of drugsMajor neurodegenerative disordersMajor neurodegenerative diseasesNon-CNS drugsFuture CNSCNS diseaseFailure rateCNS disordersCNS targetsBasic research findingsField of neuropharmacologyCNS drugsClinical developmentNeuronal functionNeurological disordersNeurodegenerative disordersNeurodegenerative diseasesHigh failure rateDrug discoveryCNSNew drugsDrugs
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