Reversal of Disease-Related Pathologies in the Fragile X Mouse Model by Selective Activation of GABA B Receptors with Arbaclofen

LTP and LTD

Malenka, R. C., & Bear, M. F. (2004) · Neuron · doi:10.1016/j.neuron.2004.09.012 · Cited by 3,968

Identification of a gene (FMR-1) containing a CGG repeat coincident with a breakpoint cluster region exhibiting length variation in fragile X syndrome

Verkerk, A. J. M. H., Pieretti, M., Sutcliffe, J. S., Fu, Y.-H., Kuhl, D. P. A., Pizzuti, A., Reiner, O., Richards, S., Victoria, M. F., Zhang, F., et al. (1991) · Cell · doi:10.1016/0092-8674(91)90397-h · Cited by 3,481

FMRP Stalls Ribosomal Translocation on mRNAs Linked to Synaptic Function and Autism

Darnell, J. C., Van Driesche, S. J., Zhang, C., Hung, K. Y. S., Mele, A., Fraser, C. E., Stone, E. F., Chen, C., Fak, J. J., Chi, S. W., et al. (2011) · Cell · doi:10.1016/j.cell.2011.06.013 · Cited by 2,072

Culturing hippocampal neurons

Kaech, S., & Banker, G. A. (2006) · Nature Protocols · doi:10.1038/nprot.2006.356 · Cited by 1,766

The mGluR theory of fragile X mental retardation

Bear, M. F., Huber, K. M., & Warren, S. T. (2004) · Trends in Neurosciences · doi:10.1016/j.tins.2004.04.009 · Cited by 1,580

Altered synaptic plasticity in a mouse model of fragile X mental retardation

Huber, K. M., Gallagher, S. M., Warren, S. T., & Bear, M. F. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.122205699 · Cited by 1,349

Morphometric study of human cerebral cortex development

Huttenlocher, P. R. (1990) · Neuropsychologia · doi:10.1016/0028-3932(90)90031-i · Cited by 1,239

Culturing hippocampal neurons.

Tanaka, H. (2002) · Folia Pharmacologica Japonica · doi:10.1254/fpj.119.163 · Cited by 1,141

Kinesin Transports RNA

Kanai, Y., Dohmae, N., & Hirokawa, N. (2004) · Neuron · doi:10.1016/j.neuron.2004.07.022 · Cited by 1,109

Fmr1 knockout mice: A model to study fragile X mental retardation

THEDUTCHBELGIANFRAGILEXCONSOR (1994) · Cell · doi:10.1016/0092-8674(94)90569-x · Cited by 1,108

Keeping the Balance: GABAB Receptors in the Developing Brain and Beyond

Bassetti, D. (2022) · Brain Sciences · doi:10.3390/brainsci12040419 · Cited by 47

From De Novo Mutations to Personalized Therapeutic Interventions in Autism

Brandler, W. M., & Sebat, J. L. (2015) · Annual Review of Medicine · doi:10.1146/annurev-med-091113-024550 · Cited by 43

Missense mutation of Fmr1 results in impaired AMPAR-mediated plasticity and socio-cognitive deficits in mice

Prieto, M., Folci, A., Poupon, G., Schiavi, S., Buzzelli, V., Pronot, M., François, U., Pousinha, P. A., Lattuada, N., Abélanet, S., et al. (2021) · Nature Communications · doi:10.1038/s41467-021-21820-1 · Cited by 46

GABABreceptor‐mediated feed‐forward circuit dysfunction in the mouse model of fragile X syndrome

Wahlstrom‐Helgren, S., & Klyachko, V. A. (2015) · The Journal of Physiology · doi:10.1113/jp271190 · Cited by 40

New Experimental Treatments for Core Social Domain in Autism Spectrum Disorders

Canitano, R. (2014) · Frontiers in Pediatrics · doi:10.3389/fped.2014.00061 · Cited by 42

Repurposing available drugs for neurodevelopmental disorders: The fragile X experience

Tranfaglia, M. R., Thibodeaux, C., Mason, D. J., Brown, D., Roberts, I., Smith, R. J., Guilliams, T., & Cogram, P. (2018) · Neuropharmacology · doi:10.1016/j.neuropharm.2018.05.004 · Cited by 41

Lithium: A Promising Treatment for Fragile X Syndrome

Liu, Z., & Smith, C. B. (2014) · ACS Chemical Neuroscience · doi:10.1021/cn500077p · Cited by 39

Autism and EMF? Plausibility of a pathophysiological link – Part I

Herbert, M. R., & Sage, C. (2013) · Pathophysiology · doi:10.1016/j.pathophys.2013.08.001 · Cited by 37

Features of the broader autism phenotype in people with epilepsy support shared mechanisms between epilepsy and autism spectrum disorder

Richard, A., Scheffer, I. E., & Wilson, S. J. (2017) · Neuroscience & Biobehavioral Reviews · doi:10.1016/j.neubiorev.2016.12.036 · Cited by 37

The promising trajectory of autism therapeutics discovery

Silverman, J. L., & Crawley, J. N. (2013) · Drug Discovery Today · doi:10.1016/j.drudis.2013.12.007 · Cited by 35

EEG as a translational biomarker and outcome measure in fragile X syndrome

Kenny, A., Wright, D., & Stanfield, A. C. (2022) · Translational Psychiatry · doi:10.1038/s41398-022-01796-2 · Cited by 36

Molecular Biomarkers in Fragile X Syndrome

Zafarullah, M., & Tassone, F. (2019) · Brain Sciences · doi:10.3390/brainsci9050096 · Cited by 36

Intervention in the Context of Development: Pathways Toward New Treatments

Veenstra-VanderWeele, J., & Warren, Z. (2015) · Neuropsychopharmacology · doi:10.1038/npp.2014.232 · Cited by 34

Twenty years of discoveries emerging from mouse models of autism

Crawley, J. N. (2023) · Neuroscience & Biobehavioral Reviews · doi:10.1016/j.neubiorev.2023.105053 · Cited by 33

Clinical potential, safety, and tolerability of arbaclofen in the treatment of autism spectrum disorder

Frye, R. E. (2014) · Drug Healthcare and Patient Safety · doi:10.2147/dhps.s39595 · Cited by 36

Fragile X syndrome neurobiology translates into rational therapy

Braat, S., & Kooy, R. F. (2014) · Drug Discovery Today · doi:10.1016/j.drudis.2014.01.013 · Cited by 35

Elevated de novo protein synthesis in FMRP-deficient human neurons and its correction by metformin treatment

Utami, K. H., Yusof, N. A. B. M., Kwa, J. E., Peteri, U., Castrén, M. L., & Pouladi, M. A. (2020) · Molecular Autism · doi:10.1186/s13229-020-00350-5 · Cited by 34

GABAB Receptor Agonist R-Baclofen Reverses Altered Auditory Reactivity and Filtering in the Cntnap2 Knock-Out Rat

Möhrle, D., Wang, W., Whitehead, S. N., & Schmid, S. (2021) · Frontiers in Integrative Neuroscience · doi:10.3389/fnint.2021.710593 · Cited by 32

FMRP regulates an ethanol-dependent shift in GABABR function and expression with rapid antidepressant properties

Wolfe, S. A., Workman, E. R., Heaney, C. F., Niere, F., Namjoshi, S. V., Cacheaux, L. P., Farris, S. P., Drew, M. R., Zemelman, B. V., Harris, R. A., et al. (2016) · Nature Communications · doi:10.1038/ncomms12867 · Cited by 31

Excessive proteostasis contributes to pathology in fragile X syndrome

Louros, S. R., Seo, S. S., Maio, B., Martinez‐Gonzalez, C., Gonzalez‐Lozano, M. A., Muscas, M., Verity, N. C., Wills, J. C., Li, K. W., Nolan, M. F., et al. (2022) · Neuron · doi:10.1016/j.neuron.2022.11.012 · Cited by 32

Pharmacotherapy for Fragile X Syndrome: Progress to Date

Davenport, M. H., Schaefer, T. L., Friedmann, K., Fitzpatrick, S. E., & Erickson, C. A. (2016) · Drugs · doi:10.1007/s40265-016-0542-y · Cited by 33

Baclofen as an adjuvant therapy for autism: a randomized, double-blind, placebo-controlled trial

Mahdavinasab, S.-M., Saghazadeh, A., Motamed-Gorji, N., Vaseghi, S., Mohammadi, M.-R., Alikhani, R., & Akhondzadeh, S. (2019) · European Child & Adolescent Psychiatry · doi:10.1007/s00787-019-01333-5 · Cited by 32

An assessment of the translational relevance of Drosophila in drug discovery

Papanikolopoulou, K., Mudher, A. K., & Skoulakis, E. M. C. (2019) · Expert Opinion on Drug Discovery · doi:10.1080/17460441.2019.1569624 · Cited by 32

Modelling fragile X syndrome in the laboratory setting: A behavioral perspective

Melancia, F., & Trezza, V. (2018) · Behavioural Brain Research · doi:10.1016/j.bbr.2018.04.042 · Cited by 31

Towards the convergent therapeutic potential of G protein‐coupled receptors in autism spectrum disorders

Annamneedi, A., Gora, C., Dudas, A., Leray, X., Bozon, V., Crépieux, P., & Pellissier, L. P. (2023) · British Journal of Pharmacology · doi:10.1111/bph.16216 · Cited by 29

GABAB receptor upregulates fragile X mental retardation protein expression in neurons

Zhang, W., Xu, C., Tu, H., Wang, Y., Sun, Q., Hu, P., Hu, Y., Rondard, P., & Liu, J. (2015) · Scientific Reports · doi:10.1038/srep10468 · Cited by 29

New insights into the molecular pathophysiology of fragile X syndrome and therapeutic perspectives from the animal model

Busquets-García, A., Maldonado, R. J., & Ozaita, A. (2014) · The International Journal of Biochemistry & Cell Biology · doi:10.1016/j.biocel.2014.05.004 · Cited by 28

Fragile X syndrome: Lessons learned from the most translated neurodevelopmental disorder in clinical trials

Duy, P. Q., & Budimirovic, D. B. (2017) · Translational Neuroscience · doi:10.1515/tnsci-2017-0002 · Cited by 28

(S)-5-(2′-Fluorophenyl)-N,N-dimethyl-1,2,3,4-tetrahydronaphthalen-2-amine, a Serotonin Receptor Modulator, Possesses Anticonvulsant, Prosocial, and Anxiolytic-like Properties in an Fmr1 Knockout Mouse Model of Fragile X Syndrome and Autism Spectrum Disorder

Armstrong, J. L., Casey, A. B., Saraf, T. S., Mukherjee, M., Booth, R. G., & Canal, C. E. (2020) · ACS Pharmacology & Translational Science · doi:10.1021/acsptsci.9b00101 · Cited by 26

Practitioner’s review: medication for children and adolescents with autism spectrum disorder (ASD) and comorbid conditions

Popow, C., Ohmann, S., & Plener, P. L. (2021) · Neuropsychiatrie · doi:10.1007/s40211-021-00395-9 · Cited by 37

Targeted pharmacological treatment of autism spectrum disorders: fragile X and Rett syndromes

Wang, H., Pati, S. B. B., Pozzo‐Miller, L. D., & Doering, L. C. (2015) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2015.00055 · Cited by 28

The FMRP/GRK4mRNA interaction uncovers a new mode of binding of the Fragile X mental retardation protein in cerebellum

Maurin, T., Melko, M., Abekhoukh, S., Khalfallah, O., Davidovic, L., Jarjat, M., D’Antoni, S., Catania, M. V., Moine, H., Bechara, E., et al. (2015) · Nucleic Acids Research · doi:10.1093/nar/gkv801 · Cited by 27

New therapeutic options for autism spectrum disorder: experimental evidences

Peñagarikano, O. (2015) · Experimental Neurobiology · doi:10.5607/en.2015.24.4.301 · Cited by 24

The GABAB receptor agonist STX209 reverses the autism‑like behaviour in an animal model of autism induced by prenatal exposure to valproic acid

Jiang, S., Xiao, L., Sun, Y., He, M., Gao, C., Zhu, C., Chang, H., Ding, J., Li, W., Wang, Y., et al. (2022) · Molecular Medicine Reports · doi:10.3892/mmr.2022.12670 · Cited by 23

Baclofen-associated neurophysiologic target engagement across species in fragile X syndrome

Jonak, C. R., Pedapati, E. V., Schmitt, L., Assad, S. A., Sandhu, M. S., DeStefano, L. A., Ethridge, L. E., Razak, K. A., Sweeney, J. A., Binder, D. K., et al. (2022) · Journal of Neurodevelopmental Disorders · doi:10.1186/s11689-022-09455-9 · Cited by 23

Neuregulin 1 Deficiency Modulates Adolescent Stress-Induced Dendritic Spine Loss in a Brain Region-Specific Manner and Increases Complement 4 Expression in the Hippocampus

Clarke, D. J., Chohan, T. W., Kassem, M. S., Smith, K. L., Chesworth, R., Karl, T., Kuligowski, M., Fok, S., Bennett, M. R., & Arnold, J. C. (2018) · Schizophrenia Bulletin · doi:10.1093/schbul/sby029 · Cited by 22

Effects of the sigma-1 receptor agonist blarcamesine in a murine model of fragile X syndrome: neurobehavioral phenotypes and receptor occupancy

Reyes, S. T., Deacon, R. M. J., Guo, S. G., Altimiras, F. J., Castillo, J. B., van der Wildt, B., Morales, A. P., Park, J. H., Klamer, D., Rosenberg, J., et al. (2021) · Scientific Reports · doi:10.1038/s41598-021-94079-7 · Cited by 22

GABAB Receptors and Cognitive Processing in Health and Disease

Vlachou, S. (2021) · Current topics in behavioral neurosciences · doi:10.1007/7854_2021_231 · Cited by 23

Therapeutic approaches for shankopathies

Wang, X., Bey, A. L., Chung, L., Krystal, A. D., & Jiang, Y. (2013) · Developmental Neurobiology · doi:10.1002/dneu.22084 · Cited by 23

An “Omic” Overview of Fragile X Syndrome

Dionne, O., & Corbin, F. (2021) · Biology · doi:10.3390/biology10050433 · Cited by 24

Curation of causal interactions mediated by genes associated with autism accelerates the understanding of gene-phenotype relationships underlying neurodevelopmental disorders

Iannuccelli, M., Vitriolo, A., Licata, L., Surdo, P. L., Contino, S., Cheroni, C., Capocefalo, D., Castagnoli, L., Testa, G., Cesareni, G., et al. (2023) · Molecular Psychiatry · doi:10.1038/s41380-023-02317-3 · Cited by 15

Challenges in developing drugs for pediatric CNS disorders: A focus on psychopharmacology

Grabb, M. C., & Gobburu, J. V. S. (2016) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2016.05.003 · Cited by 21

GABAergic dysfunction in pediatric neuro-developmental disorders

Smith‐Hicks, C. L. (2013) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2013.00269 · Cited by 21

mTOR-Dependent Spine Dynamics in Autism

Chaudry, S., & Vasudevan, N. (2022) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2022.877609 · Cited by 23

Understanding copy number variations through their genes: a molecular view on 16p11.2 deletion and duplication syndromes

Leone, R., Zuglian, C., Brambilla, R., & Morella, I. (2024) · Frontiers in Pharmacology · doi:10.3389/fphar.2024.1407865 · Cited by 22

Social Communication is an Emerging Target for Pharmacotherapy in Autism Spectrum Disorder - A Review of the Literature on Potential Agents.

Baribeau, D. A., & Anagnostou, E. A. (2014) · Europe PMC (PubMed Central) · Cited by 19

Advances in the Understanding of the Gabaergic Neurobiology of FMR1 Expanded Alleles Leading to Targeted Treatments for Fragile X Spectrum Disorder

Lozano, R., Martínez‐Cerdeño, V., & Hagerman, R. J. (2015) · Current Pharmaceutical Design · doi:10.2174/1381612821666150914121038 · Cited by 19

Regional rates of brain protein synthesis are unaltered in dexmedetomidine sedated young men with fragile X syndrome: A L-[1-11C]leucine PET study

Schmidt, K., Loutaev, I., Quezado, Z., Sheeler, C. A., & Smith, C. B. (2020) · Neurobiology of Disease · doi:10.1016/j.nbd.2020.104978 · Cited by 21

Flurothyl-induced seizure paradigm revealed higher seizure susceptibility in middle-aged Angelman syndrome mouse model

Egawa, K., Nakakubo, S., Kimura, S., Goto, T., Manabe, A., & Shiraishi, H. (2021) · Brain and Development · doi:10.1016/j.braindev.2020.12.011 · Cited by 18

Disease-Targeted Treatment Translation in Fragile X Syndrome as a Model for Neurodevelopmental Disorders

Berry‐Kravis, E. (2022) · Journal of Child Neurology · doi:10.1177/08830738221089740 · Cited by 19

Effects of acute administration of the GABA(B) receptor agonist baclofen on behavioral flexibility in rats

Beas, B. S., Setlow, B., & Bizon, J. L. (2016) · Psychopharmacology · doi:10.1007/s00213-016-4321-y · Cited by 18

Drug-responsive autism phenotypes in the 16p11.2 deletion mouse model: a central role for gene-environment interactions

Mitchell, E. J., Thomson, D. M., Openshaw, R. L., Bristow, G. C., Dawson, N., Pratt, J. A., & MORRIS, B. J. (2020) · Scientific Reports · doi:10.1038/s41598-020-69130-8 · Cited by 21

Paradoxical effect of baclofen on social behavior in the fragile X syndrome mouse model

Zeidler, S., Pop, A. S., Jaafar, I. A., de Boer, H., Buijsen, R. A. M., de Esch, C. E. F., Nieuwenhuizen‐Bakker, I., Hukema, R. K., & Willemsen, R. (2018) · Brain and Behavior · doi:10.1002/brb3.991 · Cited by 17

Translational endpoints in fragile X syndrome

de Esch, C. E. F., Zeidler, S., & Willemsen, R. (2013) · Neuroscience & Biobehavioral Reviews · doi:10.1016/j.neubiorev.2013.10.012 · Cited by 16

Meeting at the crossroads: common mechanisms in Fragile X and Down syndrome

Chang, K. T., Ro, H., Wang, W., & Min, K. (2013) · Trends in Neurosciences · doi:10.1016/j.tins.2013.08.007 · Cited by 17

Moving Toward Integrative, Multidimensional Research in Modern Psychiatry: Lessons Learned From Fragile X Syndrome

Fung, L. K., & Reiss, A. L. (2015) · Biological Psychiatry · doi:10.1016/j.biopsych.2015.12.015 · Cited by 17

Mechanisms regulating input-output function and plasticity of neurons in the absence of FMRP

Booker, S. A., & Kind, P. C. (2021) · Brain Research Bulletin · doi:10.1016/j.brainresbull.2021.06.025 · Cited by 16

Deciphering discord: How Drosophila research has enhanced our understanding of the importance of FMRP in different spatial and temporal contexts

Weisz, E. D., Monyak, R. E., & Jongens, T. A. (2015) · Experimental Neurology · doi:10.1016/j.expneurol.2015.05.015 · Cited by 16

Prenatal GABAB Receptor Agonist Administration Corrects the Inheritance of Autism-Like Core Behaviors in Offspring of Mice Prenatally Exposed to Valproic Acid

Jiang, S., He, M., Xiao, L., Sun, Y., Ding, J., Li, W., Guo, B., Wang, L., Wang, Y., Gao, C., et al. (2022) · Frontiers in Psychiatry · doi:10.3389/fpsyt.2022.835993 · Cited by 19

GABAA Alpha 2,3 Modulation Improves Select Phenotypes in a Mouse Model of Fragile X Syndrome

Schaefer, T. L., Ashworth, A. A., Tiwari, D., Tomasek, M. P., Parkins, E. V., White, A. R., Snider, A., Davenport, M. H., Grainger, L. M., Becker, R. A., et al. (2021) · Frontiers in Psychiatry · doi:10.3389/fpsyt.2021.678090 · Cited by 15

Neurodevelopment and early pharmacological interventions in Fragile X Syndrome

Milla, L. A., López‐Corral, L., Rivera, J., Zúñiga, N., Pino, G., Nunez-Parra, A., & Rio, C. A. C.-D. (2023) · Frontiers in Neuroscience · doi:10.3389/fnins.2023.1213410 · Cited by 15

New Therapeutic Options for Fragile X Syndrome

Jalnapurkar, I. R., Cochran, D., & Frazier, J. A. (2019) · Current Treatment Options in Neurology · doi:10.1007/s11940-019-0551-8 · Cited by 15

Haploinsufficiency of Tsc2 Leads to Hyperexcitability of Medial Prefrontal Cortex via Weakening of Tonic GABAB Receptor-mediated Inhibition

Bassetti, D., Lombardi, A., Kirischuk, S., & Luhmann, H. J. (2020) · Cerebral Cortex · doi:10.1093/cercor/bhaa187 · Cited by 15

Targeted treatments in fragile X syndrome

Hare, E. B., Hagerman, R. J., & Lozano, R. (2014) · Expert Opinion on Orphan Drugs · doi:10.1517/21678707.2014.903795 · Cited by 14

Is ‘bench-to-bedside’ realistic for autism? An integrative neuroscience approach

Bauman, M. D., & Schumann, C. M. (2013) · Neuropsychiatry · doi:10.2217/npy.13.18 · Cited by 13

Rates of protein synthesis are reduced in peripheral blood mononuclear cells (PBMCs) from fragile X individuals

Dionne, O., Lortie, A., Gagnon, F., & Corbin, F. (2021) · PLoS ONE · doi:10.1371/journal.pone.0251367 · Cited by 13

An Update on Psychopharmacological Treatment of Autism Spectrum Disorder

Aishworiya, R., Valica, T., Hagerman, R. J., & Restrepo, B. (2024) · FOCUS The Journal of Lifelong Learning in Psychiatry · doi:10.1176/appi.focus.24022006 · Cited by 16

Fragile X Syndrome: Lessons Learned and What New Treatment Avenues Are on the Horizon

Hagerman, R. J., & Hagerman, P. J. (2021) · The Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev-pharmtox-052120-090147 · Cited by 12

Evaluating the effect of R-Baclofen and LP-211 on autistic behavior of the BTBR and Fmr1-KO mouse models

Sharghi, S., Flunkert, S., Daurer, M., Rabl, R., Chagnaud, B. P., Leopoldo, M., Lacivita, E., Hutter‐Paier, B., & Prokesch, M. (2023) · Frontiers in Neuroscience · doi:10.3389/fnins.2023.1087788 · Cited by 12

A blueprint for research on Shankopathies: A view from research on autism spectrum disorder

Carbonetto, S. (2013) · Developmental Neurobiology · doi:10.1002/dneu.22150 · Cited by 13

Modelling Protein Synthesis as A Biomarker in Fragile X Syndrome Patient-Derived Cells

Pal, R., & Bhattacharya, A. (2019) · Brain Sciences · doi:10.3390/brainsci9030059 · Cited by 15

A MEG Study of Acute Arbaclofen (STX-209) Administration

Roberts, T. P. L., Bloy, L., Blaskey, L. G., Kuschner, E. S., Gaetz, L., Anwar, A., Ku, M., DiPiero, M. A., Bennett, A. E., & Edgar, J. C. (2019) · Frontiers in Integrative Neuroscience · doi:10.3389/fnint.2019.00069 · Cited by 13

Clinically-probed mechanisms of action in fragile-X syndrome fail to normalize translational EEG phenotypes in Fmr1 knockout mice

Janz, P., Bainier, M., Marashli, S., Gross, S., & Redondo, R. L. (2025) · Neuropharmacology · doi:10.1016/j.neuropharm.2024.110182 · Cited by 11

Intellectual and developmental disabilities research centers: Fifty years of scientific accomplishments

Walkley, S. U., Abbeduto, L., Batshaw, M. L., Bhattacharyya, A., Bookheimer, S. Y., Christian, B. T., Constantino, J. N., de Vellis, J., Doherty, D., Nelson, D. L., et al. (2019) · Annals of Neurology · doi:10.1002/ana.25531 · Cited by 11

The pediatric psychopharmacology of autism spectrum disorder: A systematic review - Part II: The future

Persico, A. M., Asta, L., Chehbani, F., Mirabelli, S., Parlatini, V., Cortese, S., Arango, C., & Vitiello, B. (2024) · Progress in Neuro-Psychopharmacology and Biological Psychiatry · doi:10.1016/j.pnpbp.2024.111176 · Cited by 11

The mGluR Theory of Fragile X: From Mice to Men

Stoppel, L. J., Osterweil, E. K., & Bear, M. F. (2017) · Elsevier eBooks · doi:10.1016/b978-0-12-804461-2.00009-3 · Cited by 9

Impact on GABA systems in monogenetic developmental CNS disorders: Clues to symptomatic treatment

Benke, D., & Möhler, H. (2017) · Neuropharmacology · doi:10.1016/j.neuropharm.2017.07.030 · Cited by 10

Neurobehavioral features and targeted treatments in fragile X syndrome: Current insights and future directions

Budimirovic, D. B., & Duy, P. Q. (2015) · Engrami · doi:10.5937/engrami1504005b · Cited by 9

Synaptic Plasticity, a Prominent Contributor to the Anxiety in Fragile X Syndrome

Yang, T., Zhao, H., Lu, C., Li, X., Xie, Y., Fu, H., & Xu, H. (2016) · Neural Plasticity · doi:10.1155/2016/9353929 · Cited by 9

Role of FMRP in rapid antidepressant effects and synapse regulation

Heaney, C. F., Namjoshi, S. V., Uneri, A., Bach, E. C., Weiner, J. L., & Raab‐Graham, K. F. (2021) · Molecular Psychiatry · doi:10.1038/s41380-020-00977-z · Cited by 10

Mice and men show the way

Anagnostou, E. A. (2012) · Nature · doi:10.1038/491196a · Cited by 8

Progress toward therapeutic potential for AFQ056 in Fragile X syndrome

Doering, L. C., Sourial, M. M., & Cheng, C. (2013) · Journal of Experimental Pharmacology · doi:10.2147/jep.s27044 · Cited by 8

Natural Products Altering GABAergic Transmission

Banerjee, S., Manisha, C., Murugan, D., & Justin, A. (2021) · IntechOpen eBooks · doi:10.5772/intechopen.99500 · Cited by 8

Lifting the Mood on Treating Fragile X

Osterweil, E. K., Kind, P. C., & Bear, M. F. (2012) · Biological Psychiatry · doi:10.1016/j.biopsych.2012.09.017 · Cited by 7

From Discovery to Innovative Translational Approaches in 80 Years of Fragile X Syndrome Research

van der Lei, M. B., & Kooy, R. F. (2025) · Biomedicines · doi:10.3390/biomedicines13040805 · Cited by 6

Emerging autism and Fragile X syndrome treatments

Parkhill, M., Salmaso, N., D’Angiulli, A., Lee, V., & Aguilar‐Valles, A. (2025) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2025.02.004 · Cited by 7

Translational Medicine Strategies in Drug Development for Neurodevelopmental Disorders

Srivastava, S., Şahin, M., & Prock, L. A. (2019) · Handbook of behavioral neuroscience · doi:10.1016/b978-0-12-803161-2.00022-9 · Cited by 6

A Differential Effect of Lovastatin versus Simvastatin in Neurodevelopmental Disorders

Muscas, M., Seo, S. S., Louros, S. R., & Osterweil, E. K. (2020) · eNeuro · doi:10.1523/eneuro.0162-20.2020 · Cited by 6

Developmental hearing loss–induced perceptual deficits are rescued by genetic restoration of cortical inhibition

Masri, S., Mowery, T. M., Fair, R., & Sanes, D. H. (2024) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2311570121 · Cited by 8

Molecular medicine of fragile X syndrome: based on known molecular mechanisms

Luo, S.-Y., Wu, L., & Duan, R. (2015) · World Journal of Pediatrics · doi:10.1007/s12519-015-0052-0 · Cited by 8

Towards Preclinical Validation of Arbaclofen (R-baclofen) Treatment for 16p11.2 Deletion SyndromePreprint

Gundersen, B. B., O’Brien, W. T., Schaffler, M. D., Schultz, M. N., Tsukahara, T., Lorenzo, S. M., Nalesso, V., Clayton, A. L., Abel, T. G., Crawley, J. N., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.05.01.538987 · Cited by 8

Review of Salient Investigational Drugs for the Treatment of Fragile X Syndrome

Munshi, K. R., Pawlowski, K. G., Gonzalez–Heydrich, J., & Picker, J. D. (2017) · Journal of Child and Adolescent Psychopharmacology · doi:10.1089/cap.2016.0200 · Cited by 5

Challenges in Translating Therapeutic Frontiers in Clinical Trials: Where Are We Now and What's Next?

Budimirovic, D. B., & Duy, P. Q. (2017) · Madridge Journal of Neuroscience · doi:10.18689/mjns-1000101 · Cited by 5

Decoding the brain's excitatory–inhibitory metabolite balance in relation to sensory responsivity and autistic traits

Chen, C., Tsai, S., Tahamata, V. M., Chuang, Y.-H., Cheng, Y., & Fan, Y. (2025) · NeuroImage · doi:10.1016/j.neuroimage.2025.121470 · Cited by 4

Human pluripotent stem cell (hPSC)-derived models for autism spectrum disorder drug discovery

Nie, L., Irwin, C. L., Geahchan, S., & Singh, K. K. (2024) · Expert Opinion on Drug Discovery · doi:10.1080/17460441.2024.2416484 · Cited by 4

Overview of Targeted Double-Blind, Placebo-Controlled Clinical Trials in Fragile X Syndrome

Ligsay, A., Hagerman, R. J., & Berry‐Kravis, E. (2017) · Elsevier eBooks · doi:10.1016/b978-0-12-804461-2.00019-6 · Cited by 4

Challenges in Conducting Clinical Trials for Pharmacotherapies in Fragile X Syndrome: Lessons Learned

Harkins, C. M., Dominick, K. C., Wink, L. K., Pedapati, E. V., Shaffer, R. C., Fitzpatrick, S. E., Davenport, M. H., Sweeney, J. A., & Erickson, C. A. (2017) · Pharmaceutical Medicine · doi:10.1007/s40290-017-0199-1 · Cited by 4

Unique and independent role of the GABAB1 subunit in embryo implantation and uterine decidualization in mice

Chen, W., Zhang, Q., Wang, H., Tan, D., & Tan, Y. (2019) · Genes & Diseases · doi:10.1016/j.gendis.2019.06.005 · Cited by 4

Pharmacological Treatments for Fragile X Syndrome Based on Synaptic Dysfunction

Telias, M. (2019) · Current Pharmaceutical Design · doi:10.2174/1381612825666191102165206 · Cited by 4

Neurobiology of Autism Spectrum Disorders

Martins, G. J. (2017) · Springer eBooks · doi:10.1007/978-3-319-42713-3_2 · Cited by 4

R-Baclofen Reverses a Social Behavior Deficit and Elevated Protein Synthesis in a Mouse Model of Fragile X Syndrome

Qin, M., Huang, T., Kader, M., Krych, L., Xia, Z., Burlin, T., Zeidler, Z., Zhao, T., & Smith, C. B. (2015) · International Journal of Neuropsychopharmacology · doi:10.1093/ijnp/pyv034 · Cited by 62

Rescue of Synaptic Phenotypes and Spatial Memory in Young Fragile X Mice

Sun, M., Hongpaisan, J., & Alkon, D. L. (2016) · Journal of Pharmacology and Experimental Therapeutics · doi:10.1124/jpet.115.231100 · Cited by 17

Potential therapeutic interventions for fragile X syndrome

Levenga, J., de Vrij, F. M. S., Oostra, B. A., & Willemsen, R. (2010) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2010.08.005 · Cited by 90

Gaboxadol Normalizes Behavioral Abnormalities in a Mouse Model of Fragile X Syndrome

Cogram, P., Deacon, R. M. J., Warner‐Schmidt, J. L., von Schimmelmann, M. J., Abrahams, B. S., & During, M. J. (2019) · Frontiers in Behavioral Neuroscience · doi:10.3389/fnbeh.2019.00141 · Cited by 53

Mechanism-Based Treatments in Neurodevelopmental Disorders: Fragile X Syndrome

Berry‐Kravis, E. (2013) · Pediatric Neurology · doi:10.1016/j.pediatrneurol.2013.12.001 · Cited by 86