ReviewDrugs2016
Pharmacotherapy for Fragile X Syndrome: Progress to Date.
Review in Drugs, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
17 citing papers in PubMed, 32 citations in OpenAlex.
- Molecular biomarkers predictive of sertraline treatment response in young children with fragile X syndrome.Brain & development · 2017Trial
- The development of early social cognitive skills in neurogenetic syndromes associated with autism: Cornelia de Lange, fragile X and Rubinstein-Taybi syndromes.Orphanet journal of rare diseases · 2021Article
- Metabolomic and lipidomic characterization of an X-chromosome deletion disorder in neural progenitor cells by UHPLC-HRMS.Journal of mass spectrometry and advances in the clinical lab · 2021Article
- The Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities.Frontiers in psychiatry · 2021Article
- An Observational Study of Social Interaction Skills and Behaviors in Cornelia de Lange, Fragile X and Rubinstein-Taybi Syndromes.Journal of autism and developmental disorders · 2020Observational
- Transcriptome signature analysis repurposes trifluoperazine for the treatment of fragile X syndrome in mouse model.Communications biology · 2020Article
- Fragile X syndrome clinical trials: exploring parental decision-making.Journal of intellectual disability research : JIDR · 2019Article
- Brain Penetrable Histone Deacetylase 6 Inhibitor SW-100 Ameliorates Memory and Learning Impairments in a Mouse Model of Fragile X Syndrome.ACS chemical neuroscience · 2019Article
- New Therapeutic Options for Fragile X Syndrome.Current treatment options in neurology · 2019Review
- Roles for Arc in metabotropic glutamate receptor-dependent LTD and synapse elimination: Implications in health and disease.Seminars in cell & developmental biology · 2018Review
- Drug development for neurodevelopmental disorders: lessons learned from fragile X syndrome.Nature reviews. Drug discovery · 2018Review
- Fragile X syndrome: a review of clinical and molecular diagnoses.Italian journal of pediatrics · 2017Review
- A New Link Between Insulin Signaling and Fragile X Syndrome.Neuroscience bulletin · 2017Article
- Updated report on tools to measure outcomes of clinical trials in fragile X syndrome.Journal of neurodevelopmental disorders · 2017Review
- Fragile X targeted pharmacotherapy: lessons learned and future directions.Journal of neurodevelopmental disorders · 2017Review
- Review
- Imbalance between Glutamate and GABA in Fmr1 Knockout Astrocytes Influences Neuronal Development.Genes · 2016Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
To date, no drug is approved for the treatment of Fragile X Syndrome (FXS) although many drugs are used to manage challenging behaviors from a symptomatic perspective in this population. While our understanding of FXS pathophysiology is expanding, efforts to devise targeted FXS-specific treatments have had limited success in placebo-controlled trials. Compounds aimed at rectifying excessive glutamate and deficient gamma-aminobutyric acid (GABA) neurotransmission, as well as other signaling pathways known to be affected by Fragile X Mental Retardation Protein (FMRP) are under various phases of development in FXS. With the failure of several metabotropic glutamate receptor subtype 5 (mGlur5) selective antagonists under clinical investigation, no clear single treatment appears to be greatly effective. These recent challenges call into question various aspects of clinical study design in FXS. More objective outcome measures are under development and validation. Future trials will likely be aimed at correcting multiple pathways known to be disrupted by the loss of FMRP. This review offers a brief summary of the prevalence, phenotypic characteristics, genetic causes and molecular functions of FMRP in the brain (as these have been extensively reviewed elsewhere), discusses the most recent finding in FXS drug development, and summarizes FXS trials utilizing symptomatic treatment.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.