Paul Wang, MD
About
Biography
Dr. Wang is a developmental-behavioral pediatrician who has worked in academia, large pharma, start-up biotech, and the non-profit sector. Since 2002, his career has focused on drug development, especially for autism and related genetic disorders. He led pioneering trials for Fragile X syndrome, 16p11.2 deletion syndrome, and autism. Currently at the Simons Foundation, he continues to focus on trial design, the development and validation of outcome measures for autism, and biomarker strategy for autism drug development. He is a member of the federally-appointed Interagency Autism Coordinating Committee, and has served in leadership positions for the American Academy of Pediatrics, the American Board of Pediatrics, and other professional groups.
Departments & Organizations
Education & Training
- MD
- Yale School of Medicine (1986)
- AB
- Harvard College (1982)
Research
Research at a Glance
Yale Co-Authors
Publications Timeline
Stuart Alan Weinzimer, MD
Publications
2021
Clinical trial strategies for rare neurodevelopmental disorders: challenges and opportunities
Krishnan M, Berry-Kravis E, Capal J, Carpenter R, Gringras P, Hipp J, Miller M, Mingorance A, Philpot B, Pletcher M, Rotenberg A, Tjeertes J, Wang P, Willgoss T, de Wit M, Jeste S. Clinical trial strategies for rare neurodevelopmental disorders: challenges and opportunities. Nature Reviews Drug Discovery 2021, 20: 653-654. PMID: 34002058, DOI: 10.1038/d41573-021-00085-9.Peer-Reviewed Original ResearchMeSH Keywords and ConceptsA white paper on a neurodevelopmental framework for drug discovery in autism and other neurodevelopmental disorders
Díaz-Caneja C, State M, Hagerman R, Jacquemont S, Marín O, Bagni C, Umbricht D, Simonoff E, de Andrés-Trelles F, Kaale A, Pandina G, Gómez-Mancilla B, Wang P, Cusak J, Siafis S, Leucht S, Parellada M, Loth E, Charman T, Buitelaar J, Murphy D, Arango C. A white paper on a neurodevelopmental framework for drug discovery in autism and other neurodevelopmental disorders. European Neuropsychopharmacology 2021, 48: 49-88. PMID: 33781629, DOI: 10.1016/j.euroneuro.2021.02.020.Peer-Reviewed Original Research
2018
Implicit Learning in Children and Adults With Williams Syndrome
Don A, Schellenberg E, Reber A, DiGirolamo K, Wang P. Implicit Learning in Children and Adults With Williams Syndrome. 2018, 201-225. DOI: 10.4324/9780203764800-9.Peer-Reviewed Original ResearchConceptsImplicit learningWilliams syndromeComparison groupEvidence of implicit learningRotor pursuit taskNonverbal intelligenceWorking memoryGroup of childrenWS groupGroup differencesLearning paradigmChronological ageIndependent of ageImplicitTaskAdultsIQLearningDevelopmental processesMemoryChildrenParticipantsParadigmWilliamGroup
2017
Drug development for neurodevelopmental disorders: lessons learned from fragile X syndrome
Berry-Kravis E, Lindemann L, Jønch A, Apostol G, Bear M, Carpenter R, Crawley J, Curie A, Des Portes V, Hossain F, Gasparini F, Gomez-Mancilla B, Hessl D, Loth E, Scharf S, Wang P, Von Raison F, Hagerman R, Spooren W, Jacquemont S. Drug development for neurodevelopmental disorders: lessons learned from fragile X syndrome. Nature Reviews Drug Discovery 2017, 17: 280-299. PMID: 29217836, PMCID: PMC6904225, DOI: 10.1038/nrd.2017.221.Peer-Reviewed Original ResearchMeSH Keywords and ConceptsConceptsFragile X mental retardation protein 1X syndromeNeurodevelopmental disorder associated with intellectual disabilityDisorder associated with intellectual disabilityMetabotropic glutamate receptor 5Fmr1 knockout miceAssessment of CognitionAutism spectrum disorderGlutamate receptor 5Fragile X syndromeNeurobiological mechanismsIntellectual disabilityBehavioral measuresBehavioral phenotypesSpectrum disorderNeurodevelopmental disordersSynaptic plasticityCore phenotypeGABA receptorsDevelopmental outcomesClinical trialsYoung childrenCognitionSecondary behaviorDisordersAdaptive behavior in autism: Minimal clinically important differences on the Vineland‐II
Chatham C, Taylor K, Charman T, D'ardhuy X, Eule E, Fedele A, Hardan A, Loth E, Murtagh L, del Valle Rubido M, San Jose Caceres A, Sevigny J, Sikich L, Snyder L, Tillmann J, Ventola P, Walton‐Bowen K, Wang P, Willgoss T, Bolognani F. Adaptive behavior in autism: Minimal clinically important differences on the Vineland‐II. Autism Research 2017, 11: 270-283. PMID: 28941213, PMCID: PMC5997920, DOI: 10.1002/aur.1874.Peer-Reviewed Original ResearchArbaclofen in fragile X syndrome: results of phase 3 trials
Berry-Kravis E, Hagerman R, Visootsak J, Budimirovic D, Kaufmann W, Cherubini M, Zarevics P, Walton-Bowen K, Wang P, Bear M, Carpenter R. Arbaclofen in fragile X syndrome: results of phase 3 trials. Journal Of Neurodevelopmental Disorders 2017, 9: 3. PMID: 28616094, PMCID: PMC5467054, DOI: 10.1186/s11689-016-9181-6.Peer-Reviewed Original ResearchConceptsClinical Global Impression-ImprovementFragile X syndromeClinical Global Impression-SeveritySocial avoidanceABC-CFXAberrant Behavior Checklist-Community EditionHigh dose groupAnimal models of fragile X syndromeX syndromeModel of fragile X syndromeVineland Adaptive Behavior ScalesEffect sizeFlexible dose trialSerotonin reuptake inhibitorsSocial Avoidance subscaleChild studiesAdaptive Behavior ScalesAdolescent/adult studyFixed dose trialsDose groupParenting Stress IndexPhase 3 placebo-controlled trialsPreclinical models to clinical trialsResults of phase 3 trialsDose trial
2016
Arbaclofen in Children and Adolescents with Autism Spectrum Disorder: A Randomized, Controlled, Phase 2 Trial
Veenstra-VanderWeele J, Cook E, King B, Zarevics P, Cherubini M, Walton-Bowen K, Bear M, Wang P, Carpenter R. Arbaclofen in Children and Adolescents with Autism Spectrum Disorder: A Randomized, Controlled, Phase 2 Trial. Neuropsychopharmacology 2016, 42: 1390-1398. PMID: 27748740, PMCID: PMC5436109, DOI: 10.1038/npp.2016.237.Peer-Reviewed Original ResearchMeSH Keywords and ConceptsConceptsAutism spectrum disorderFragile X syndromeSpectrum disorderClinician-rated Clinical Global ImpressionAnimal models of fragile X syndromeGABA-BModel of fragile X syndromeSocial avoidance symptomsClinical Global ImpressionPlacebo-controlledGABA-B agonistSubgroups of individualsPilot open-label trialExploratory post hoc analysisAffective labilityAvoidance symptomsPost hoc analysis of participantsOpen-label trialBehavioral phenotypesGlobal ImpressionArbaclofenSecondary analysisX syndromeSocial behaviorSocial domains
2014
Measuring social communication behaviors as a treatment endpoint in individuals with autism spectrum disorder
Anagnostou E, Jones N, Huerta M, Halladay A, Wang P, Scahill L, Horrigan J, Kasari C, Lord C, Choi D, Sullivan K, Dawson G. Measuring social communication behaviors as a treatment endpoint in individuals with autism spectrum disorder. Autism 2014, 19: 622-636. PMID: 25096930, DOI: 10.1177/1362361314542955.Peer-Reviewed Original ResearchMeSH Keywords and ConceptsConceptsAutism spectrum disorderSpectrum disorderSymptom domainsEarly indicator of autism spectrum disorderMeasures of social communicationSocial communicationIndicator of autism spectrum disorderSocial communication impairmentsSocial communication measuresSocial communication deficitsSocial communication behaviorsCore deficitCommunication deficitsCommunication impairmentsAutism SpeaksPsychometric propertiesOutcome measuresAutismCommunication measuresTreatment targetCommunication behaviorsTreatment approachesDisordersMonthly conference callsTreatment endpoint
2013
STX209 (Arbaclofen) for Autism Spectrum Disorders: An 8-Week Open-Label Study
Erickson C, Veenstra-Vanderweele J, Melmed R, McCracken J, Ginsberg L, Sikich L, Scahill L, Cherubini M, Zarevics P, Walton-Bowen K, Carpenter R, Bear M, Wang P, King B. STX209 (Arbaclofen) for Autism Spectrum Disorders: An 8-Week Open-Label Study. Journal Of Autism And Developmental Disorders 2013, 44: 958-964. PMID: 24272415, DOI: 10.1007/s10803-013-1963-z.Peer-Reviewed Original ResearchMeSH Keywords and ConceptsConceptsAutism spectrum disorderSpectrum disorderClinical Global Impression ScaleNon-syndromic autism spectrum disorderExcitatory to inhibitory neurotransmissionGlobal Impression ScaleSocial Responsiveness ScaleGABA-B agonistFragile X syndromeABC-irritabilityImpression ScaleWithdrawal subscalePlacebo-controlled studyAutistic disorderResponse scaleSTX209GABA-BX syndromeOpen-label studyInhibitory neurotransmissionArbaclofenDisordersSubscalesEvidence of benefitOpen-label
2012
Effects of STX209 (Arbaclofen) on Neurobehavioral Function in Children and Adults with Fragile X Syndrome: A Randomized, Controlled, Phase 2 Trial
Berry-Kravis E, Hessl D, Rathmell B, Zarevics P, Cherubini M, Walton-Bowen K, Mu Y, Nguyen D, Gonzalez-Heydrich J, Wang P, Carpenter R, Bear M, Hagerman R. Effects of STX209 (Arbaclofen) on Neurobehavioral Function in Children and Adults with Fragile X Syndrome: A Randomized, Controlled, Phase 2 Trial. Science Translational Medicine 2012, 4: 152ra127. PMID: 22993294, DOI: 10.1126/scitranslmed.3004214.Peer-Reviewed Original ResearchMeSH Keywords and ConceptsConceptsFragile X syndromeAberrant Behavior Checklist-IrritabilityX syndromeAvoidance ScaleAnimal models of fragile X syndromeSymptoms of fragile X syndromeModel of fragile X syndromeNeurobehavioral functionSevere social impairmentImprove social functioningGABA(B) agonistGlobal measuresPlacebo-controlled crossover studyVisual analog scale ratingsPost hoc subgroupsSocial impairmentProblem behaviorsBeneficial treatment effectsSTX209Raw scoresSocial functioningPost hoc analysisIncidence of sedationDouble-blindFMR1 gene