Evidence map›Paper›PMID 38385885›Full record

ReviewDevelopmental medicine and child neurology2024

State-of-the-art therapies for fragile X syndrome.

Dragana Protic, Randi Hagerman

Open access · bronzeAbstract readReview
In one paragraph

Review in Developmental medicine and child neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
9.9field-weighted citation impact, top 2% 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

9 citing papers in PubMed, 18 citations in OpenAlex.

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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

2 authors at 2 institutions in 2 countries.

Dragana ProticDepartment of Pharmacology, Clinical Pharmacology and Toxicology, Faculty of Medicine University of Belgrade, Belgrade, Serbia.ORCID 0000-0002-2137-5405
Randi HagermanMedical Investigation of Neurodevelopmental Disorders Institute, University of California, Davis, CA, USA.ORCID 0000-0001-5029-8448
NeuroDevelopment Center · USUnited Cerebral Palsy · US

Funding

GENOTYPE/PHENOTYPE RELATIONSHIPS IN FRAGILE X FAMILIESR01HD036071 · NICHD · UNIVERSITY OF CALIFORNIA DAVIS · PI PAUL J HAGERMAN, RANDI J. HAGERMAN · 1998 to 2026
$13.8M
Azrieli FoundationNICHD NIH HHS R01 HD036071the National Institute of Child Health and Human Development HD036071the Science Fund of the Republic of Serbia Profram DIASPORA, Grant No 6431806the Science Fund of the Republic of Serbia Program IDEA, Grant No 7673781the Victor E. LaFave III Memorial Fund
6 · The paper itself

Abstract

Fragile X syndrome (FXS) is a neurodevelopmental disorder caused by a full mutation (> 200 CGG repeats) in the FMR1 gene. FXS is the leading cause of inherited intellectual disabilities and the most commonly known genetic cause of autism spectrum disorder. Children with FXS experience behavioral and sleep problems, anxiety, inattention, learning difficulties, and speech and language delays. There are no approved medications for FXS; however, there are several interventions and treatments aimed at managing the symptoms and improving the quality of life of individuals with FXS. A combination of non-pharmacological therapies and pharmacotherapy is currently the most effective treatment for FXS. Currently, several targeted treatments, such as metformin, sertraline, and cannabidiol, can be used by clinicians to treat FXS. Gene therapy is rapidly developing and holds potential as a prospective treatment option. Soon its efficacy and safety in patients with FXS will be demonstrated. WHAT THIS PAPER ADDS: Targeted treatment of fragile X syndrome (FXS) is the best current therapeutic approach. Gene therapy holds potential as a prospective treatment for FXS in the future.

Indexed as

Fragile X SyndromeGenetic TherapyFragile X Messenger Ribonucleoprotein 1HumansFragile X Messenger Ribonucleoprotein 1

Identifiers

PMID38385885
PMCPMC11144093
OpenAlexW4392040529

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.