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

Author: BearMark F, Berry-KravisElizabeth M, CarpenterRandall L, CherubiniMaryann, Gonzalez-HeydrichJoseph, HagermanRandi J, HesslDavid, MuYi, NguyenDanh V, RathmellBarbara, Walton-BowenKaren, WangPaul P, ZarevicsPeter

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

A study was conducted to investigate the potential benefits of STX209, a GABA(B) agonist, for treating fragile X syndrome. The study included 63 participants with fragile X syndrome and found no significant improvement in irritability compared to placebo. However, positive trends were observed in parent-nominated problem behaviors and significant improvements were seen in social function, particularly in a subgroup with more severe social impairment. The treatment was well-tolerated with sedation and headache as the most common side effects.
Paper Details　
Original Abstract of the Article :
Research on animal models of fragile X syndrome suggests that STX209, a γ-aminobutyric acid type B (GABA(B)) agonist, might improve neurobehavioral function in affected patients. We evaluated whether STX209 improves behavioral symptoms of fragile X syndrome in a randomized, double-blind, placebo-con...See full text at original site
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引用元：
https://doi.org/10.1126/scitranslmed.3004214

データ提供：米国国立医学図書館（NLM）

A New Path for Fragile X Syndrome

Fragile X syndrome is a genetic condition affecting cognitive development and social behavior. It's like a desert oasis that is slowly drying up, making it difficult for individuals to thrive. This study investigates the potential of a drug called STX209 to improve some of the symptoms of Fragile X syndrome.

Hope for the Oasis

The researchers found that while STX209 did not improve irritability in individuals with Fragile X syndrome, it showed promise in improving social function and behavior. It's like finding a new source of water in the desert, offering hope for a brighter future. The study found that individuals with more severe social impairments showed the greatest improvements.

Finding a Sustainable Solution

This study is a step forward in understanding how to manage the symptoms of Fragile X syndrome. It's like discovering a new path in the desert, leading towards a more fulfilling life. It highlights the need for continued research to develop effective treatments for this complex condition.

Dr. Camel's Conclusion

This research is a reminder that even in the face of challenges, there is always hope for finding new pathways to improvement. It's a journey of discovery, much like exploring a vast desert, where each step brings us closer to a brighter future.

Date :
  1. Date Completed 2013-06-27
  2. Date Revised 2013-11-21
Further Info :

Pubmed ID

22993294

DOI: Digital Object Identifier

10.1126/scitranslmed.3004214

Related Literature

Article Analysis
・Effective treatment of fragile x syndrome
SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

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