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Shopbop Designer Fashion Brands. Warehouse Deals Open-Box Discounts. Amazon Renewed Like-new products you can trust. Received Nov 14; Accepted Nov This article has been cited by other articles in PMC. Abstract Fragile X syndrome FXS is the leading known cause of inherited intellectual disability and autism spectrum disorder.
Clinical Development of Targeted Fragile X Syndrome Treatments: An Industry Perspective
Keywords: fragile X syndrome, clinical trials, targeted treatments, drug development. Introduction Fragile X syndrome FXS is the most common inherited cause of intellectual disability and the second most common cause of intellectual disability after Down syndrome [ 1 , 2 ]. Open in a separate window. Development of Targeted Therapies 4. Clinical Development FXS has been at the forefront of efforts to test preclinical evidence for targeted interventions in clinical studies. Phase Compound Identifier Industry-Sponsored? Considerations for Future Preclinical Research The failure of animal models to predict the efficacy of potential therapies in psychiatric diseases and neurologic disorders, including FXS, is a central problem in drug development.
Considerations for Conducting Clinical Trials As previous experience has shown, clinical trials to evaluate potential FXS pharmacologic treatments are challenging. Table 3 Desirable clinical outcome measure attributes [ 76 ]. Conclusions There remains a great need for safe and effective treatments for FXS, particularly for targeted treatments that surpass symptom management and address the pathophysiologic abnormalities that underlie the most common manifestations.
Author Contributions All authors A. Funding This research received no external funding. Conflicts of Interest A. References 1.
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