Evidence map›Paper›PMID 24050745›Full record

ReviewCurrent clinical pharmacology2013

Evaluation of current pharmacological treatment options in the management of Rett syndrome: from the present to future therapeutic alternatives.

Christopher A Chapleau, Jane Lane, Lucas Pozzo-Miller, Alan K Percy

Open access · bronzeAbstract readReview
In one paragraph

Review in Current clinical pharmacology, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed, 1 pooled it
2.8field-weighted citation impact, top 9% of its field
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

18 citing papers in PubMed, 1 synthesis or guideline pooled it, 35 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Rett syndrome.Nature reviews. Disease primers · 2024
    Review
  4. Article
  5. Review
  6. Article
  7. Article
  8. Article
  9. Drug Studies on Rett Syndrome: From Bench to Bedside.Journal of autism and developmental disorders · 2020
    Review
  10. Article
  11. Review
  12. Genetic Approaches to Understanding Psychiatric Disease.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2017
    Review
  13. Article
  14. Therapeutic Potential of Transcranial Focused Ultrasound for Rett Syndrome.Medical science monitor : international medical journal of experimental and clinical research · 2016
    Article
  15. Article
  16. PTP1B inhibition suggests a therapeutic strategy for Rett syndrome.The Journal of clinical investigation · 2015
    Article
  17. Review
  18. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors at 1 institution in 1 country.

Christopher A ChapleauDepartment of Pediatrics, CIRC-320, The University of Alabama at Birmingham, 1530 3rd Avenue South, Birmingham, AL 35294-0021, USA. chris.a.chapleau@gmail.com.
Jane Lane
Lucas Pozzo-Miller
Alan K Percy
University of Alabama at Birmingham · US

Funding

Training and Career Development ComponentU54HD061222 · NICHD · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI PERCY, ALAN KENNETH · 2009 to 2019
$12.4M
UAB MENTAL RETARDATION RESEARCH CENTER (MRRC)P30HD038985 · NICHD · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI BRENNER, MICHAEL · 2000 to 2012
$8.6M
ACTIONS OF BDNF ON CA2+ SIGNALS IN HIPPOCAMPAL NEURONSR01NS040593 · NINDS · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI POZZO-MILLER, LUCAS D · 2000 to 2010
$2.8M
MeCP2 Modulation of BDNF Signaling: Shared Mechanisms of Rett and AutismR01NS065027 · NINDS · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI POZZO-MILLER, LUCAS D · 2010 to 2014
$1.7M
Role of BDNF in dendritic pathologies caused by Rett-associated MeCP2 mutationsR21NS057780 · NINDS · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI POZZO-MILLER, LUCAS D · 2007 to 2008
$400k
NICHD NIH HHS HD061222NICHD NIH HHS HD38985NICHD NIH HHS P30 HD038985NICHD NIH HHS U54 HD061222NINDS NIH HHS NS057780NINDS NIH HHS NS065027NINDS NIH HHS NS40593NINDS NIH HHS R01 NS040593NINDS NIH HHS R01 NS065027NINDS NIH HHS R21 NS057780
6 · The paper itself

Abstract

Neurodevelopmental disorders are a large family of conditions of genetic or environmental origin that are characterized by deficiencies in cognitive and behavioral functions. The therapeutic management of individuals with these disorders is typically complex and is limited to the treatment of specific symptoms that characterize each disorder. The neurodevelopmental disorder Rett syndrome (RTT) is the leading cause of severe intellectual disability in females. Mutations in the gene encoding the transcriptional regulator methyl-CpG-binding protein 2 (MECP2), located on the X chromosome, have been confirmed in more than 95% of individuals meeting diagnostic criteria for classical RTT. RTT is characterized by an uneventful early infancy followed by stagnation and regression of growth, motor, language, and social skills later in development. This review will discuss the genetics, pathology, and symptoms that distinguish RTT from other neurodevelopmental disorders associated with intellectual disability. Because great progress has been made in the basic and clinical science of RTT, the goal of this review is to provide a thorough assessment of current pharmacotherapeutic options to treat the symptoms associated with this disorder. Furthermore, we will highlight recent discoveries made with novel pharmacological interventions in experimental preclinical phases, and which have reversed pathological phenotypes in mouse and cell culture models of RTT and may result in clinical trials.

Indexed as

Drug DesignAnimalsDisease Models, AnimalFemaleHumansMethyl-CpG-Binding Protein 2MiceMutationPhenotypeRett SyndromeMethyl-CpG-Binding Protein 2

Identifiers

PMID24050745
PMCPMC3789853
OpenAlexW2169572802

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

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.