Effects of AFQ056 on language learning in fragile X syndrome,The Journal of Clinical Investigation

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Effects of AFQ056 on language learning in fragile X syndrome
The Journal of Clinical Investigation ( IF 15.9 ) Pub Date : 2023 , DOI: 10.1172/jci171723
Elizabeth Berry-Kravis ₁ , Leonard Abbeduto ₂ , Randi Hagerman ₃ , Christopher S Coffey ₄ , Merit Cudkowicz ₅ , Craig A Erickson ₆ , Andrea McDuffie ₂ , David Hessl ₂ , Lauren Ethridge ₇ , Flora Tassone ₈ , Walter E Kaufmann ₉ , Katherine Friedmann ₁₀ , Lauren Bullard ₂ , Anne Hoffmann ₁₁ , Jeremy Veenstra-VanderWeele ₁₂ , Kevin Staley ₅ , David Klements ₅ , Michael Moshinsky ₅ , Brittney Harkey ₅ , Jeff Long ₄ , Janel Fedler ₄ , Elizabeth Klingner ₄ , Dixie Ecklund ₄ , Michele Costigan ₄ , Trevis Huff ₄ , Brenda Pearson ₄ ,

Affiliation

Departments of Pediatrics, Neurological Sciences, and Anatomy & Cell Biology, Rush University Medical Center, Chicago, Illinois, USA.
MIND Institute and Department of Psychiatry and Behavioral Sciences and.
MIND Institute and Department of Pediatrics, UCD, Sacramento, California, USA.
Department of Biostatistics, University of Iowa, Iowa City, Iowa, USA.
Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA.
Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Department of Psychology, University of Oklahoma, Norman, Oklahoma, and Department of Pediatrics, University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma, USA.
MIND Institute and Department of Biochemistry and Molecular Medicine, UCD, Sacramento, California, USA.
Department of Human Genetics, Emory University School of Medicine, Atlanta, Georgia, USA.
College of Nursing and.
Departments of Pediatrics and Communication Disorders and Sciences, Rush University Medical Center, Chicago, Illinois, USA.
Department of Psychiatry, Columbia University, and New York State Psychiatric Institute, New York, New York, USA.

BACKGROUND. FXLEARN, the first-ever large multisite trial of effects of disease-targeted pharmacotherapy on learning, was designed to explore a paradigm for measuring effects of mechanism-targeted treatment in fragile X syndrome (FXS). In FXLEARN, the effects of metabotropic glutamate receptor type 5 (mGluR5) negative allosteric modulator (NAM) AFQ056 on language learning were evaluated in 3- to 6-year-old children with FXS, expected to have more learning plasticity than adults, for whom prior trials of mGluR5 NAMs have failed.

中文翻译：

AFQ056 对脆性 X 综合征语言学习的影响

背景。FXLEARN 是有史以来第一个针对疾病靶向药物治疗对学习影响的大型多中心试验，旨在探索测量脆性 X 综合征 (FXS) 机制靶向治疗效果的范例。在 FXLEARN 中，代谢型谷氨酸受体 5 型 (mGluR5) 负变构调节剂 (NAM) AFQ056 对 3 至 6 岁 FXS 儿童语言学习的影响进行了评估，预计这些儿童比成人具有更多的学习可塑性。 mGluR5 NAM 的先前试验失败了。

更新日期：2024-03-02

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