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 success