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