Fragile X targeted pharmacotherapy: lessons learned and future directions

Pharmacotherapy for Fragile X Syndrome: Progress to Date

(2016) Matthew H. Davenport et al.   DRUGS

Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials

(2016) Elizabeth Berry-Kravis et al.   Science Translational Medicine

Clinical trials for neurodevelopmental disorders: At a therapeutic frontier

(2016) Shafali S. Jeste et al.   Science Translational Medicine

Reduced habituation of auditory evoked potentials indicate cortical hyper-excitability in Fragile X Syndrome

(2016) L E Ethridge et al.   Translational Psychiatry

Endocannabinoid-mediated improvement on a test of aversive memory in a mouse model of fragile X syndrome

(2015) Mei Qin et al.   BEHAVIOURAL BRAIN RESEARCH

Expression of fragile X mental retardation protein in neurons and glia of the developing and adult mouse brain

(2015) Shervin Gholizadeh et al.   BRAIN RESEARCH

The GABAAreceptor is an FMRP target with therapeutic potential in fragile X syndrome

(2015) Sien Braat et al.   CELL CYCLE

Age-Dependent Long-Term Potentiation Deficits in the Prefrontal Cortex of theFmr1Knockout Mouse Model of Fragile X Syndrome

(2015) Henry G. S. Martin et al.   CEREBRAL CORTEX

Mavoglurant in adolescents with fragile X syndrome: analysis of Clinical Global Impression-Improvement source data from a double-blind therapeutic study followed by an open-label, long-term extension study

(2015) Donald B. Bailey et al.   Journal of Neurodevelopmental Disorders

Homer Protein-Metabotropic Glutamate Receptor Binding Regulates Endocannabinoid Signaling and Affects Hyperexcitability in a Mouse Model of Fragile X Syndrome

(2015) A.-H. Tang et al.   JOURNAL OF NEUROSCIENCE

NNZ-2566, a Novel Analog of (1–3) IGF-1, as a Potential Therapeutic Agent for Fragile X Syndrome

(2015) Robert M. J. Deacon et al.   NEUROMOLECULAR MEDICINE

Selective Role of the Catalytic PI3K Subunit p110β in Impaired Higher Order Cognition in Fragile X Syndrome

(2015) Christina Gross et al.   Cell Reports

Increased Expression of the PI3K Enhancer PIKE Mediates Deficits in Synaptic Plasticity and Behavior in Fragile X Syndrome

(2015) Christina Gross et al.   Cell Reports

Effect of lovastatin on behavior in children and adults with fragile X syndrome: An open-label study

(2014) Artuela Çaku et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Cholesterol levels in Fragile X syndrome

(2014) Elizabeth Berry-Kravis et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Analysis ofFmr1Deletion in a Subpopulation of Post-Mitotic Neurons in Mouse Cortex and Hippocampus

(2014) Anahita Amiri et al.   Autism Research

Quantitative Phosphoproteomics of Murine Fmr1-KO Cell Lines Provides New Insights into FMRP-Dependent Signal Transduction Mechanisms

(2014) Katarina Matic et al.   JOURNAL OF PROTEOME RESEARCH

Impact of acamprosate on plasma amyloid-β precursor protein in youth: A pilot analysis in fragile X syndrome-associated and idiopathic autism spectrum disorder suggests a pharmacodynamic protein marker

(2014) Craig A. Erickson et al.   JOURNAL OF PSYCHIATRIC RESEARCH

Activity-dependent alterations in the sensitivity to BDNF-TrkB signaling may promote excessive dendritic arborization and spinogenesis in fragile X syndrome in order to compensate for compromised postsynaptic activity

(2014) Sang Woo Kim et al.   MEDICAL HYPOTHESES

Fragile X Syndrome: A Review of Associated Medical Problems

(2014) S. A. Kidd et al.   PEDIATRICS

High MMP-9 activity levels in fragile X syndrome are lowered by minocycline

(2013) Magdalena Dziembowska et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Glycogen Synthase Kinase-3 Inhibitors Reverse Deficits in Long-term Potentiation and Cognition in Fragile X Mice

(2013) Aimee V. Franklin et al.   BIOLOGICAL PSYCHIATRY

A Randomized Double-Blind, Placebo-Controlled Trial of Minocycline in Children and Adolescents with Fragile X Syndrome

(2013) Mary Jacena S. Leigh et al.   JOURNAL OF DEVELOPMENTAL AND BEHAVIORAL PEDIATRICS

Electrocortical changes associated with minocycline treatment in fragile X syndrome

(2013) Andrea Schneider et al.   JOURNAL OF PSYCHOPHARMACOLOGY

From FMRP Function to Potential Therapies for Fragile X Syndrome

(2013) Ferzin Sethna et al.   NEUROCHEMICAL RESEARCH

Mechanism of Action for NNZ-2566 Anti-inflammatory Effects Following PBBI Involves Upregulation of Immunomodulator ATF3

(2013) Casandra M. Cartagena et al.   NEUROMOLECULAR MEDICINE

Lovastatin Corrects Excess Protein Synthesis and Prevents Epileptogenesis in a Mouse Model of Fragile X Syndrome

(2013) Emily K. Osterweil et al.   NEURON

Long-lasting effects of minocycline on behavior in young but not adult Fragile X mice

(2013) L.E. Dansie et al.   NEUROSCIENCE

Impact of acamprosate on behavior and brain-derived neurotrophic factor: an open-label study in youth with fragile X syndrome

(2013) Craig A. Erickson et al.   PSYCHOPHARMACOLOGY

Resilience to audiogenic seizures is associated with p-ERK1/2 dephosphorylation in the subiculum of Fmr1 knockout mice

(2013) Giulia Curia et al.   Frontiers in Cellular Neuroscience

Pharmacological treatment of fragile X syndrome with GABAergic drugs in a knockout mouse model

(2012) Inge Heulens et al.   BEHAVIOURAL BRAIN RESEARCH

Effectiveness and Safety of Donepezil in Boys With Fragile X Syndrome

(2012) Jitendra Kumar Sahu et al.   JOURNAL OF CHILD NEUROLOGY

Activation of the extracellular signal-regulated kinase pathway contributes to the behavioral deficit of fragile x-syndrome

(2012) Xinglong Wang et al.   JOURNAL OF NEUROCHEMISTRY

Evidence of a Distinct Behavioral Phenotype in Young Boys With Fragile X Syndrome and Autism

(2012) Jason J. Wolff et al.   JOURNAL OF THE AMERICAN ACADEMY OF CHILD AND ADOLESCENT PSYCHIATRY

Trajectories of Early Brain Volume Development in Fragile X Syndrome and Autism

(2012) Heather Cody Hazlett et al.   JOURNAL OF THE AMERICAN ACADEMY OF CHILD AND ADOLESCENT PSYCHIATRY

Genetic Removal of p70 S6 Kinase 1 Corrects Molecular, Synaptic, and Behavioral Phenotypes in Fragile X Syndrome Mice

(2012) Aditi Bhattacharya et al.   NEURON

Chronic Pharmacological mGlu5 Inhibition Corrects Fragile X in Adult Mice

(2012) Aubin Michalon et al.   NEURON

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

(2012) C. Henderson et al.   Science Translational Medicine

Uncoupling of the endocannabinoid signalling complex in a mouse model of fragile X syndrome

(2012) Kwang-Mook Jung et al.   Nature Communications

Molecular Mechanisms of Fragile X Syndrome: A Twenty-Year Perspective

(2011) Michael R. Santoro et al.   Annual Review of Pathology-Mechanisms of Disease

Management of familial hypercholesterolemia in children and young adults: Consensus paper developed by a panel of lipidologists, cardiologists, paediatricians, nutritionists, gastroenterologists, general practitioners and a patient organization

(2011) O.S. Descamps et al.   ATHEROSCLEROSIS

Genetic reduction of group 1 metabotropic glutamate receptors alters select behaviors in a mouse model for fragile X syndrome

(2011) Alexia M. Thomas et al.   BEHAVIOURAL BRAIN RESEARCH

What Can We Learn about Autism from Studying Fragile X Syndrome?

(2011) Dejan B. Budimirovic et al.   DEVELOPMENTAL NEUROSCIENCE

Lithium reverses increased rates of cerebral protein synthesis in a mouse model of fragile X syndrome

(2011) Zhong-Hua Liu et al.   NEUROBIOLOGY OF DISEASE

Effects of intranasal oxytocin on social anxiety in males with fragile X syndrome

(2011) Scott S. Hall et al.   PSYCHONEUROENDOCRINOLOGY

Epigenetic Modification of the FMR1 Gene in Fragile X Syndrome Is Associated with Differential Response to the mGluR5 Antagonist AFQ056

(2011) S. Jacquemont et al.   Science Translational Medicine

Open-label add-on treatment trial of minocycline in fragile X syndrome

(2010) Carlo Paribello et al.   BMC Neurology

Open-label riluzole in fragile X syndrome

(2010) Craig A. Erickson et al.   BRAIN RESEARCH

Pharmacological reversal of synaptic plasticity deficits in the mouse model of Fragile X syndrome by group II mGluR antagonist or lithium treatment

(2010) Catherine H. Choi et al.   BRAIN RESEARCH

NMDA receptor hypofunction in the dentate gyrus and impaired context discrimination in adult Fmr1 knockout mice

(2010) Brennan D. Eadie et al.   HIPPOCAMPUS

Lithium ameliorates phenotypic deficits in a mouse model of fragile X syndrome

(2010) Zhong-Hua Liu et al.   INTERNATIONAL JOURNAL OF NEUROPSYCHOPHARMACOLOGY

Loss of the Fragile X Mental Retardation Protein Decouples Metabotropic Glutamate Receptor Dependent Priming of Long-Term Potentiation From Protein Synthesis

(2010) Benjamin D. Auerbach et al.   JOURNAL OF NEUROPHYSIOLOGY

Excess Phosphoinositide 3-Kinase Subunit Synthesis and Activity as a Novel Therapeutic Target in Fragile X Syndrome

(2010) C. Gross et al.   JOURNAL OF NEUROSCIENCE

Dysregulation of mTOR Signaling in Fragile X Syndrome

(2010) A. Sharma et al.   JOURNAL OF NEUROSCIENCE

Hypersensitivity to mGluR5 and ERK1/2 Leads to Excessive Protein Synthesis in the Hippocampus of a Mouse Model of Fragile X Syndrome

(2010) E. K. Osterweil et al.   JOURNAL OF NEUROSCIENCE

Defective GABAergic Neurotransmission and Pharmacological Rescue of Neuronal Hyperexcitability in the Amygdala in a Mouse Model of Fragile X Syndrome

(2010) J. L. Olmos-Serrano et al.   JOURNAL OF NEUROSCIENCE

Enhanced Endocannabinoid Signaling Elevates Neuronal Excitability in Fragile X Syndrome

(2010) L. Zhang et al.   JOURNAL OF NEUROSCIENCE

Fragile X mice: Reduced long-term potentiation and N-Methyl-D-Aspartate receptor-mediated neurotransmission in dentate gyrus

(2010) Sung Hwan Yun et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Cholinergic dysfunction in fragile X syndrome and potential intervention: A preliminary1H MRS study

(2009) Shelli R. Kesler et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Lithium ameliorates altered glycogen synthase kinase-3 and behavior in a mouse model of Fragile X syndrome

(2009) Christopher J. Yuskaitis et al.   BIOCHEMICAL PHARMACOLOGY

A pilot open label, single dose trial of fenobam in adults with fragile X syndrome

(2009) E Berry-Kravis et al.   JOURNAL OF MEDICAL GENETICS

NNZ-2566 treatment inhibits neuroinflammation and pro-inflammatory cytokine expression induced by experimental penetrating ballistic-like brain injury in rats

(2009) Hans H Wei et al.   Journal of Neuroinflammation

NNZ-2566, a Glypromate Analog, Improves Functional Recovery and Attenuates Apoptosis and Inflammation in a Rat Model of Penetrating Ballistic-Type Brain Injury

(2009) Xi-Chun May Lu et al.   JOURNAL OF NEUROTRAUMA

A Placebo-Controlled, Fixed-Dose Study of Aripiprazole in Children and Adolescents With Irritability Associated With Autistic Disorder

(2009) Ronald N. Marcus et al.   JOURNAL OF THE AMERICAN ACADEMY OF CHILD AND ADOLESCENT PSYCHIATRY

Increased GABAB Receptor-Mediated Signaling Reduces the Susceptibility of Fragile X Knockout Mice to Audiogenic Seizures

(2009) L. K. K. Pacey et al.   MOLECULAR PHARMACOLOGY

Acamprosate: Recent Findings and Future Research Directions

(2008) Karl Mann et al.   ALCOHOLISM-CLINICAL AND EXPERIMENTAL RESEARCH

Co-occurring conditions associated withFMR1gene variations: Findings from a national parent survey

(2008) Donald B. Bailey et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Expression of the GABAergic system in animal models for fragile X syndrome and fragile X associated tremor/ataxia syndrome (FXTAS)

(2008) Charlotte D'Hulst et al.   BRAIN RESEARCH

The Fragile X Syndrome Protein Represses Activity-Dependent Translation through CYFIP1, a New 4E-BP

(2008) Ilaria Napoli et al.   CELL

Fragile X syndrome

(2008) Kathryn B Garber et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Open-Label Treatment Trial of Lithium to Target the Underlying Defect in Fragile X Syndrome

(2008) Elizabeth Berry-Kravis et al.   JOURNAL OF DEVELOPMENTAL AND BEHAVIORAL PEDIATRICS

Ras Signaling Mechanisms Underlying Impaired GluR1-Dependent Plasticity Associated with Fragile X Syndrome

(2008) H. Hu et al.   JOURNAL OF NEUROSCIENCE

Synaptic ionotropic glutamate receptors and plasticity are developmentally altered in the CA1 field ofFmr1knockout mice

(2008) Yair Pilpel et al.   JOURNAL OF PHYSIOLOGY-LONDON

Identification of small molecules rescuing fragile X syndrome phenotypes in Drosophila

(2008) Shuang Chang et al.   Nature Chemical Biology

Molecular mechanisms of interaction between the neuroprotective substance riluzole and GABAA-receptors

(2008) K. Jahn et al.   NAUNYN-SCHMIEDEBERGS ARCHIVES OF PHARMACOLOGY

Rescue of behavioral phenotype and neuronal protrusion morphology in Fmr1 KO mice

(2008) Femke M.S. de Vrij et al.   NEUROBIOLOGY OF DISEASE

Advances in the Treatment of Fragile X Syndrome

(2008) R. J. Hagerman et al.   PEDIATRICS

Aberrant early-phase ERK inactivation impedes neuronal function in fragile X syndrome

(2008) S. H. Kim et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA