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脆性X综合征:关于在临床试验中捕捉治疗改善情况的最新研究进展

Fragile X Syndrome: Recent Research Updates toward Capturing Treatments' Improvement in Clinical Trials.

作者信息

Budimirovic Dejan B, Protic Dragana D

机构信息

Department of Psychiatry, Fragile X Clinic, Kennedy Krieger Institute, Baltimore, MD 21205, USA.

Department of Psychiatry & Behavioral Sciences-Child Psychiatry, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.

出版信息

Brain Sci. 2022 Sep 22;12(10):1276. doi: 10.3390/brainsci12101276.

DOI:10.3390/brainsci12101276
PMID:36291209
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9599739/
Abstract

This Special Issue of nine manuscripts contribute novel data on treatment updates in fragile X syndrome (FXS) [...].

摘要

本期特刊包含九篇论文,提供了脆性X综合征(FXS)治疗进展的新数据[……]。

相似文献

1
Fragile X Syndrome: Recent Research Updates toward Capturing Treatments' Improvement in Clinical Trials.脆性X综合征:关于在临床试验中捕捉治疗改善情况的最新研究进展
Brain Sci. 2022 Sep 22;12(10):1276. doi: 10.3390/brainsci12101276.
2
Emerging pharmacologic treatment options for fragile X syndrome.脆性X综合征新出现的药物治疗选择
Appl Clin Genet. 2015 Apr 7;8:75-93. doi: 10.2147/TACG.S35673. eCollection 2015.
3
Molecular medicine of fragile X syndrome: based on known molecular mechanisms.脆性X综合征的分子医学:基于已知分子机制
World J Pediatr. 2016 Feb;12(1):19-27. doi: 10.1007/s12519-015-0052-0. Epub 2015 Nov 7.
4
Advances in the Understanding of the Gabaergic Neurobiology of FMR1 Expanded Alleles Leading to Targeted Treatments for Fragile X Spectrum Disorder.对导致脆性X谱系障碍靶向治疗的FMR1扩展等位基因的γ-氨基丁酸能神经生物学认识的进展
Curr Pharm Des. 2015;21(34):4972-4979. doi: 10.2174/1381612821666150914121038.
5
Mechanism-based treatments in neurodevelopmental disorders: fragile X syndrome.基于机制的神经发育障碍治疗方法:脆性X综合征
Pediatr Neurol. 2014 Apr;50(4):297-302. doi: 10.1016/j.pediatrneurol.2013.12.001. Epub 2013 Dec 4.
6
BDNF in fragile X syndrome.脆性 X 综合征中的脑源性神经营养因子。
Neuropharmacology. 2014 Jan;76 Pt C:729-36. doi: 10.1016/j.neuropharm.2013.05.018. Epub 2013 May 29.
7
Epigenetic modifications in human fragile X pluripotent stem cells; Implications in fragile X syndrome modeling.人类脆性X多能干细胞中的表观遗传修饰;对脆性X综合征建模的影响。
Brain Res. 2017 Feb 1;1656:55-62. doi: 10.1016/j.brainres.2015.10.004. Epub 2015 Oct 20.
8
Disease-Targeted Treatment Translation in Fragile X Syndrome as a Model for Neurodevelopmental Disorders.脆性 X 综合征的疾病靶向治疗转化作为神经发育障碍模型。
J Child Neurol. 2022 Oct;37(10-11):797-812. doi: 10.1177/08830738221089740. Epub 2022 Jul 5.
9
The challenges of clinical trials in fragile X syndrome.脆性X综合征临床试验面临的挑战。
Psychopharmacology (Berl). 2014 Mar;231(6):1237-50. doi: 10.1007/s00213-013-3289-0. Epub 2013 Oct 31.
10
Pharmacotherapy for Fragile X Syndrome: Progress to Date.脆性X综合征的药物治疗:迄今的进展
Drugs. 2016 Mar;76(4):431-45. doi: 10.1007/s40265-016-0542-y.

本文引用的文献

1
Disease-Targeted Treatment Translation in Fragile X Syndrome as a Model for Neurodevelopmental Disorders.脆性 X 综合征的疾病靶向治疗转化作为神经发育障碍模型。
J Child Neurol. 2022 Oct;37(10-11):797-812. doi: 10.1177/08830738221089740. Epub 2022 Jul 5.
2
Fragile X Syndrome: From Molecular Aspect to Clinical Treatment.脆性 X 综合征:从分子角度到临床治疗。
Int J Mol Sci. 2022 Feb 9;23(4):1935. doi: 10.3390/ijms23041935.
3
A Genotype-Phenotype Study of High-Resolution Nucleic Acid and Protein Analyses in Fragile X Patients with Neurobehavioral Assessments.一项针对伴有神经行为评估的脆性X综合征患者进行高分辨率核酸和蛋白质分析的基因型-表型研究。
Brain Sci. 2020 Sep 30;10(10):694. doi: 10.3390/brainsci10100694.
4
Cognitive Training Deep Dive: The Impact of Child, Training Behavior and Environmental Factors within a Controlled Trial of Cogmed for Fragile X Syndrome.认知训练深入探讨:在针对脆性X综合征的Cogmed对照试验中儿童、训练行为和环境因素的影响
Brain Sci. 2020 Sep 25;10(10):671. doi: 10.3390/brainsci10100671.
5
Response to Placebo in Fragile X Syndrome Clinical Trials: An Initial Analysis.脆性X综合征临床试验中的安慰剂反应:初步分析
Brain Sci. 2020 Sep 11;10(9):629. doi: 10.3390/brainsci10090629.
6
Adaptive Skills in FXS: A Review of the Literature and Evaluation of the PEDI-Computer Adaptive Test (PEDI-CAT) to Measure Adaptive Skills.脆性X综合征中的适应性技能:文献综述及用于测量适应性技能的儿童残疾评估量表计算机自适应测试(PEDI-CAT)评估
Brain Sci. 2020 Jun 6;10(6):351. doi: 10.3390/brainsci10060351.
7
Evaluating Social Interactions Using the Autism Screening Instrument for Education Planning-3rd Edition (ASIEP-3): Interaction Assessment in Children and Adults with Fragile X Syndrome.使用《教育规划自闭症筛查工具第三版》(ASIEP - 3)评估社交互动:脆性X综合征儿童和成人的互动评估
Brain Sci. 2020 Apr 22;10(4):248. doi: 10.3390/brainsci10040248.
8
Delineating Repetitive Behavior Profiles across the Lifespan in Fragile X Syndrome.描绘脆性X综合征患者一生中的重复行为特征
Brain Sci. 2020 Apr 17;10(4):239. doi: 10.3390/brainsci10040239.
9
Examination of Correlates to Health-Related Quality of Life in Individuals with Fragile X Syndrome.脆性X综合征患者健康相关生活质量的相关因素研究。
Brain Sci. 2020 Apr 4;10(4):213. doi: 10.3390/brainsci10040213.
10
Language across the Lifespan in Fragile X Syndrome: Characteristics and Considerations for Assessment.脆性X综合征患者一生的语言发展:特征与评估考量
Brain Sci. 2020 Apr 4;10(4):212. doi: 10.3390/brainsci10040212.