2017
Challenges in Conducting Clinical Trials for Pharmacotherapies in Fragile X Syndrome: Lessons Learned
Harkins C, Dominick K, Wink L, Pedapati E, Shaffer R, Fitzpatrick S, Davenport M, Sweeney J, Erickson C. Challenges in Conducting Clinical Trials for Pharmacotherapies in Fragile X Syndrome: Lessons Learned. Pharmaceutical Medicine 2017, 31: 235-244. DOI: 10.1007/s40290-017-0199-1.Peer-Reviewed Original ResearchAutism spectrum disorderFragile X syndromeBehavioral outcome measuresClinician-reported instrumentsUse of parentsBehavioral measuresEye gazeSpectrum disorderNeuroscientific advancesReport instrumentX syndromeTreatment developmentCommon single gene causeBrain functionRater biasBehavioral heterogeneityIntellectual disabilityPharmacotherapy trialsOutcome measuresPotential explanationTreatment-associated changesQuantitative measuresMeasuresDisordersFuture successFragile X targeted pharmacotherapy: lessons learned and future directions
Erickson C, Davenport M, Schaefer T, Wink L, Pedapati E, Sweeney J, Fitzpatrick S, Brown W, Budimirovic D, Hagerman R, Hessl D, Kaufmann W, Berry-Kravis E. Fragile X targeted pharmacotherapy: lessons learned and future directions. Journal Of Neurodevelopmental Disorders 2017, 9: 7. PMID: 28616096, PMCID: PMC5467059, DOI: 10.1186/s11689-017-9186-9.Peer-Reviewed Original ResearchClinical trial rationaleCurrent prescribing practicesDrug targetsAppropriate outcome measuresClinical trial designTreatment development effortsFXS animal modelsNumerous potential drug targetsTrial rationaleGABAergic neurotransmissionPrescribing practicesSyndrome pathophysiologyClinical trialsOutcome measuresTrial designAnimal modelsDrug approachSingle gene disordersPotential drug targetsTreatment developmentPharmacotherapyDrugsPathophysiologyTrial execution
2016
Pharmacotherapy for Fragile X Syndrome: Progress to Date
Davenport M, Schaefer T, Friedmann K, Fitzpatrick S, Erickson C. Pharmacotherapy for Fragile X Syndrome: Progress to Date. Drugs 2016, 76: 431-445. PMID: 26858239, DOI: 10.1007/s40265-016-0542-y.Peer-Reviewed Original ResearchConceptsFragile X Mental Retardation ProteinGamma-aminobutyric acid (GABA) neurotransmissionMore objective outcome measuresPlacebo-controlled trialFragile X syndromeClinical study designObjective outcome measuresSymptomatic treatmentLoss of FMRPFuture trialsExcessive glutamateOutcome measuresSelective antagonistClinical investigationSingle treatmentStudy designSymptomatic perspectiveGenetic causeX syndromeMental retardation proteinTrialsDrug developmentTreatmentSyndromeDrugs