Next Article in Journal
Alterations in Motor Cortical Representation of Muscles Following Incomplete Spinal Cord Injury in Humans
Next Article in Special Issue
Early Identification of Fragile X Syndrome through Expanded Newborn Screening
Previous Article in Journal
Update on Insomnia after Mild Traumatic Brain Injury
Previous Article in Special Issue
Clinical Development of Targeted Fragile X Syndrome Treatments: An Industry Perspective
Open AccessCommentary

Best Practices in Fragile X Syndrome Treatment Development

Division of Child & Adolescent Psychiatry, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH 45229, USA
Department of Psychiatry and Behavioral Neuroscience, University of Cincinnati College of Medicine, Cincinnati, OH 45219, USA
Department of Human Genetics, Emory University School of Medicine, Atlanta, GA 30322, USA
MIND Institute, Department of Neurology, School of Medicine, University of California, Davis, CA 95817, USA
Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD 21205, USA
Behavioral Sciences-Child Psychiatry, Johns Hopkins University School of Medicine, Baltimore, MD 21287, USA
Duke Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, USA
Autism & Developmental Medicine Institute, Geisinger, Lewisburg, PA 17837, USA
National Fragile X Foundation, McLean, VA 22102, USA
MIND Institute and Department of Psychiatry and Behavioral Sciences, School of Medicine, University of California, Davis, CA 95817, USA
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;
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.

AMA Style

Erickson CA, Kaufmann WE, Budimirovic DB, Lachiewicz A, Haas-Givler B, Miller RM, Weber JD, Abbeduto L, Hessl D, Hagerman RJ, Berry-Kravis E. Best Practices in Fragile X Syndrome Treatment Development. Brain Sciences. 2018; 8(12):224.

Chicago/Turabian Style

Erickson, Craig A.; Kaufmann, Walter E.; Budimirovic, Dejan B.; Lachiewicz, Ave; Haas-Givler, Barbara; Miller, Robert M.; Weber, Jayne D.; Abbeduto, Leonard; Hessl, David; Hagerman, Randi J.; Berry-Kravis, Elizabeth. 2018. "Best Practices in Fragile X Syndrome Treatment Development" Brain Sci. 8, no. 12: 224.

Find Other Styles
Note that from the first issue of 2016, MDPI journals use article numbers instead of page numbers. See further details here.

Article Access Map by Country/Region

Search more from Scilit
Back to TopTop