Drug development for neurodevelopmental disorders: lessons learned from fragile X syndrome. (Q45957740)

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6 April 2020

review article

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title

Drug development for neurodevelopmental disorders: lessons learned from fragile X syndrome (English)

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neurodevelopmental disorder

6 April 2020

main subject

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3

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Aurore Curie

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Vincent Des Portes

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6 April 2020

author name string

Elizabeth M Berry-Kravis

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Lothar Lindemann

2

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George Apostol

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6 April 2020

Mark F Bear

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Randall L Carpenter

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6 April 2020

Jacqueline N Crawley

7

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6 April 2020

Farah Hossain

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Fabrizio Gasparini

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Baltazar Gomez-Mancilla

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6 April 2020

David Hessl

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Eva Loth

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Sebastian H Scharf

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Paul P Wang

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Florian Von Raison

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Randi Hagerman

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Will Spooren

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Sébastien Jacquemont

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publication date

8 December 2017

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Nature Reviews Drug Discovery

6 April 2020

published in

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volume

17

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issue

4

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6 April 2020

page(s)

280-299

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Instability of a 550-base pair DNA segment and abnormal methylation in fragile X syndrome

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Molecular actions and therapeutic potential of lithium in preclinical and clinical studies of CNS disorders

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

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

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Open-label treatment trial of lithium to target the underlying defect in fragile X syndrome

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Synaptic plasticity: one STEP at a time

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Taking STEPs forward to understand fragile X syndrome

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Genetic manipulation of STEP reverses behavioral abnormalities in a fragile X syndrome mouse model.

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Biochemical mechanisms for translational regulation in synaptic plasticity

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S6K1 phosphorylates and regulates fragile X mental retardation protein (FMRP) with the neuronal protein synthesis-dependent mammalian target of rapamycin (mTOR) signaling cascade

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Targeting Translation Control with p70 S6 Kinase 1 Inhibitors to Reverse Phenotypes in Fragile X Syndrome Mice.

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Endocannabinoid-mediated control of synaptic transmission

31 March 2020

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Possible Therapeutic Doses of Cannabinoid Type 1 Receptor Antagonist Reverses Key Alterations in Fragile X Syndrome Mouse Model

31 March 2020

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The endocannabinoid system and rimonabant: a new drug with a novel mechanism of action involving cannabinoid CB1 receptor antagonism--or inverse agonism--as potential obesity treatment and other therapeutic use.

31 March 2020

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Efficacy and safety of the weight-loss drug rimonabant: a meta-analysis of randomised trials

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Physiological activation of synaptic Rac>PAK (p-21 activated kinase) signaling is defective in a mouse model of fragile X syndrome

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Synaptic AMPA receptor composition in development, plasticity and disease.

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AMPA Receptors as Therapeutic Targets for Neurological Disorders

31 March 2020

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https://api.crossref.org/works/10.1038/NRD.2017.221

Pharmacology of ampakine modulators: from AMPA receptors to synapses and behavior

31 March 2020

1 reference

https://api.crossref.org/works/10.1038/NRD.2017.221

Reduced cortical synaptic plasticity and GluR1 expression associated with fragile X mental retardation protein deficiency.

31 March 2020

1 reference

https://api.crossref.org/works/10.1038/NRD.2017.221

Effect of CX516, an AMPA-modulating compound, on cognition and behavior in fragile X syndrome: a controlled trial

31 March 2020

1 reference

https://api.crossref.org/works/10.1038/NRD.2017.221

Pharmacotherapy for Fragile X Syndrome: Progress to Date.

31 March 2020

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https://api.crossref.org/works/10.1038/NRD.2017.221

Review of targeted treatments in fragile X syndrome

31 March 2020

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https://api.crossref.org/works/10.1038/NRD.2017.221

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

31 March 2020

1 reference

https://api.crossref.org/works/10.1038/NRD.2017.221

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

31 March 2020

1 reference

https://api.crossref.org/works/10.1038/NRD.2017.221

A randomized double-blind, placebo-controlled trial of minocycline in children and adolescents with fragile x syndrome

31 March 2020

1 reference

https://api.crossref.org/works/10.1038/NRD.2017.221

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

31 March 2020

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https://api.crossref.org/works/10.1038/NRD.2017.221

Cholinergic dysfunction in fragile X syndrome and potential intervention: a preliminary 1H MRS study.

31 March 2020

1 reference

https://api.crossref.org/works/10.1038/NRD.2017.221

A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome.

31 March 2020

1 reference

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Effectiveness and safety of donepezil in boys with fragile x syndrome: a double-blind, randomized, controlled pilot study

31 March 2020

1 reference

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Strengths and weaknesses in the intellectual functioning of males with fragile X syndrome.

31 March 2020

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12 December 2020

Specification of the neurobehavioral phenotype in males with fragile X syndrome

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12 December 2020

The neurocognitive phenotype of female carriers of fragile X: additional evidence for specificity.

1 reference

12 December 2020

Mental status of females with an FMR1 gene full mutation

1 reference

12 December 2020

Characterization of the anticonvulsant properties of ganaxolone (CCD 1042; 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one), a selective, high-affinity, steroid modulator of the gamma-aminobutyric acid(A) receptor

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12 December 2020

Genotype, molecular phenotype, and cognitive phenotype: correlations in fragile X syndrome

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12 December 2020

Rimonabant: suicide and depression. Depression and suicidal tendencies are about twice as frequent with rimonabant as with placebo

1 reference

12 December 2020

Effects of STX209 (arbaclofen) on neurobehavioral function in children and adults with fragile X syndrome: a randomized, controlled, phase 2 trial

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12 December 2020

Lost in translation: animal models and clinical trials in cancer treatment

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12 December 2020

Identifiers

DOI

10.1038/NRD.2017.221

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29217836 AND SRC:MED&resulttype=core&format=json

Dimensions Publication ID

6 April 2020

0 references

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29217836 AND SRC:MED&resulttype=core&format=json

6 April 2020

PubMed publication ID

29217836

1 reference