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Open AccessReview

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

1
Ovid Therapeutics Inc., New York, NY 10036, USA
2
Child Study Center, Yale University, New Haven, CT 06520, USA
3
Departments of Psychiatry and Behavioral Sciences, Kennedy Krieger Institute and Child Psychiatry, Johns Hopkins University, Baltimore, MD 21205, USA
4
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), 214; https://doi.org/10.3390/brainsci8120214
Received: 14 November 2018 / Revised: 28 November 2018 / Accepted: 30 November 2018 / Published: 5 December 2018
(This article belongs to the Special Issue Towards Mechanism-based Treatments for Fragile X Syndrome)
Fragile X syndrome (FXS) is the leading known cause of inherited intellectual disability and autism spectrum disorder. It is caused by a mutation of the fragile X mental retardation 1 (FMR1) gene, resulting in a deficit of fragile X mental retardation protein (FMRP). The clinical presentation of FXS is variable, and is typically associated with developmental delays, intellectual disability, a wide range of behavioral issues, and certain identifying physical features. Over the past 25 years, researchers have worked to understand the complex relationship between FMRP deficiency and the symptoms of FXS and, in the process, have identified several potential targeted therapeutics, some of which have been tested in clinical trials. Whereas most of the basic research to date has been led by experts at academic institutions, the pharmaceutical industry is becoming increasingly involved with not only the scientific community, but also with patient advocacy organizations, as more promising pharmacological agents are moving into the clinical stages of development. The objective of this review is to provide an industry perspective on the ongoing development of mechanism-based treatments for FXS, including identification of challenges and recommendations for future clinical trials. View Full-Text
Keywords: fragile X syndrome; clinical trials; targeted treatments; drug development fragile X syndrome; clinical trials; targeted treatments; drug development
MDPI and ACS Style

Lee, A.W.; Ventola, P.; Budimirovic, D.; Berry-Kravis, E.; Visootsak, J. Clinical Development of Targeted Fragile X Syndrome Treatments: An Industry Perspective. Brain Sci. 2018, 8, 214.

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