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Clinical Development of Targeted Fragile X Syndrome Treatments: An Industry Perspective
Open AccessCommentary

Best Practices in Fragile X Syndrome Treatment Development

1
Division of Child & Adolescent Psychiatry, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH 45229, USA
2
Department of Psychiatry and Behavioral Neuroscience, University of Cincinnati College of Medicine, Cincinnati, OH 45219, USA
3
Department of Human Genetics, Emory University School of Medicine, Atlanta, GA 30322, USA
4
MIND Institute, Department of Neurology, School of Medicine, University of California, Davis, CA 95817, USA
5
Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD 21205, USA
6
Behavioral Sciences-Child Psychiatry, Johns Hopkins University School of Medicine, Baltimore, MD 21287, USA
7
Duke Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, USA
8
Autism & Developmental Medicine Institute, Geisinger, Lewisburg, PA 17837, USA
9
National Fragile X Foundation, McLean, VA 22102, USA
10
MIND Institute and Department of Psychiatry and Behavioral Sciences, School of Medicine, University of California, Davis, CA 95817, USA
11
Departments of Pediatrics, Neurological Sciences, Biochemistry, Rush University Medical Center, Chicago, IL 60612, USA
*
Author to whom correspondence should be addressed.
Brain Sci. 2018, 8(12), 224; https://doi.org/10.3390/brainsci8120224
Received: 21 November 2018 / Revised: 10 December 2018 / Accepted: 12 December 2018 / Published: 15 December 2018
(This article belongs to the Special Issue Towards Mechanism-based Treatments for Fragile X Syndrome)
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. View Full-Text
Keywords: fragile X syndrome; clinical trials; treatment development; best practices fragile X syndrome; clinical trials; treatment development; best practices
MDPI and ACS Style

Erickson, C.A.; Kaufmann, W.E.; Budimirovic, D.B.; Lachiewicz, A.; Haas-Givler, B.; Miller, R.M.; Weber, J.D.; Abbeduto, L.; Hessl, D.; Hagerman, R.J.; Berry-Kravis, E. Best Practices in Fragile X Syndrome Treatment Development. Brain Sci. 2018, 8, 224.

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