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

Overview of attention for article published in Journal of Neurodevelopmental Disorders, August 2017
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  • Good Attention Score compared to outputs of the same age (65th percentile)

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44 Mendeley
Title
A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome
Published in
Journal of Neurodevelopmental Disorders, August 2017
DOI 10.1186/s11689-017-9207-8
Pubmed ID
Authors

Andrew Ligsay, Anke Van Dijck, Danh V. Nguyen, Reymundo Lozano, Yanjun Chen, Erika S. Bickel, David Hessl, Andrea Schneider, Kathleen Angkustsiri, Flora Tassone, Berten Ceulemans, R. Frank Kooy, Randi J. Hagerman

Abstract

Gamma-aminobutyric acid (GABA) system deficits are integral to the pathophysiologic development of fragile X syndrome (FXS). Ganaxolone, a GABAA receptor positive allosteric modulator, is hypothesized to improve symptoms such as anxiety, hyperactivity, and attention deficits in children with FXS. This study was a randomized, double-blind, placebo-controlled, crossover trial of ganaxolone in children with FXS, aged 6-17 years. Sixty-one participants were assessed for eligibility, and 59 were randomized to the study. Fifty-five participants completed at least the first arm and were included in the intention-to-treat analysis; 51 participants completed both treatment arms. There were no statistically significant improvements observed on the primary outcome measure (Clinical Global Impression-Improvement), the key secondary outcome measure (Pediatric Anxiety Rating Scale-R), or any other secondary outcome measures in the overall study population. However, post-hoc analyses revealed positive trends in areas of anxiety, attention, and hyperactivity in participants with higher baseline anxiety and low full-scale IQ scores. No serious adverse events (AEs) occurred, although there was a significant increase in the frequency and severity of AEs related to ganaxolone compared to placebo. While ganaxolone was found to be safe, there were no significant improvements in the outcome measures in the overall study population. However, ganaxolone in subgroups of children with FXS, including those with higher anxiety or lower cognitive abilities, might have beneficial effects. ClinicalTrials.gov, NCT01725152.

Twitter Demographics

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Mendeley readers

The data shown below were compiled from readership statistics for 44 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 44 100%

Demographic breakdown

Readers by professional status Count As %
Researcher 8 18%
Student > Doctoral Student 8 18%
Student > Master 8 18%
Student > Bachelor 4 9%
Student > Ph. D. Student 4 9%
Other 12 27%
Readers by discipline Count As %
Neuroscience 10 23%
Unspecified 8 18%
Psychology 7 16%
Biochemistry, Genetics and Molecular Biology 7 16%
Medicine and Dentistry 5 11%
Other 7 16%

Attention Score in Context

This research output has an Altmetric Attention Score of 4. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 04 June 2019.
All research outputs
#3,840,416
of 13,457,898 outputs
Outputs from Journal of Neurodevelopmental Disorders
#136
of 286 outputs
Outputs of similar age
#90,487
of 266,359 outputs
Outputs of similar age from Journal of Neurodevelopmental Disorders
#1
of 1 outputs
Altmetric has tracked 13,457,898 research outputs across all sources so far. This one has received more attention than most of these and is in the 71st percentile.
So far Altmetric has tracked 286 research outputs from this source. They typically receive more attention than average, with a mean Attention Score of 9.4. This one has gotten more attention than average, scoring higher than 52% of its peers.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 266,359 tracked outputs that were published within six weeks on either side of this one in any source. This one has gotten more attention than average, scoring higher than 65% of its contemporaries.
We're also able to compare this research output to 1 others from the same source and published within six weeks on either side of this one. This one has scored higher than all of them