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Brain Sci. 2018 Dec 15;8(12). pii: E224. doi: 10.3390/brainsci8120224.

Best Practices in Fragile X Syndrome Treatment Development.

Author information

1
Division of Child & Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA. craig.erickson@cchmc.org.
2
Department of Psychiatry and Behavioral Neuroscience, University of Cincinnati College of Medicine, Cincinnati, OH 45219, USA. craig.erickson@cchmc.org.
3
Department of Human Genetics, Emory University School of Medicine, Atlanta, GA 30322, USA. wekaufmann@ucdavis.edu.
4
MIND Institute, Department of Neurology, School of Medicine, University of California, Davis, CA 95817, USA. wekaufmann@ucdavis.edu.
5
Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD 21205, USA. budimirovic@kennedykrieger.org.
6
Behavioral Sciences-Child Psychiatry, Johns Hopkins University School of Medicine, Baltimore, MD 21287, USA. budimirovic@kennedykrieger.org.
7
Duke Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, USA. ave.lachiewicz@duke.edu.
8
Autism & Developmental Medicine Institute, Geisinger, Lewisburg, PA 17837, USA. bahaasgivler@geisinger.edu.
9
National Fragile X Foundation, McLean, VA 22102, USA. robby@fragilex.org.
10
National Fragile X Foundation, McLean, VA 22102, USA. jayne@fragilex.org.
11
MIND Institute and Department of Psychiatry and Behavioral Sciences, School of Medicine, University of California, Davis, CA 95817, USA. ljabbeduto@ucdavis.edu.
12
MIND Institute and Department of Psychiatry and Behavioral Sciences, School of Medicine, University of California, Davis, CA 95817, USA. drhessl@ucdavis.edu.
13
MIND Institute and Department of Psychiatry and Behavioral Sciences, School of Medicine, University of California, Davis, CA 95817, USA. rjhagerman@ucdavis.edu.
14
Departments of Pediatrics, Neurological Sciences, Biochemistry, Rush University Medical Center, Chicago, IL 60612, USA. Elizabeth_Berry-Kravis@rush.edu.

Abstract

Preclinical studies using animal models of fragile X syndrome have yielded several agents that rescue a wide variety of phenotypes. However, translation of these treatments to humans with the disorder has not yet been successful, shedding light on a variety of limitations with both animal models and human trial design. As members of the Clinical Trials Committee of the National Fragile X Foundation, we have discussed a variety of recommendations at the level of preclinical development, transition from preclinical to human projects, family involvement, and multi-site trial planning. Our recommendations are made with the vision that effective new treatment will lie at the intersection of innovation, rigorous and reproducible research, and stakeholder involvement.

KEYWORDS:

best practices; clinical trials; fragile X syndrome; treatment development

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