Guide Learn To Fly (Double Blind Study Book 1)

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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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Riley C. Thurman A. Psychiatric symptoms in boys with fragile X syndrome: A comparison with nonsyndromic autism spectrum disorder. Kaufmann W. Autism Spectrum Disorder in fragile X syndrome: Cooccurring conditions and current treatment. Grigsby J. The fragile X mental retardation 1 gene FMR1 : Historical perspective, phenotypes, mechanism, pathology, and epidemiology. Banerjee A.

Brain Res. Rajaratnam A. Fragile X syndrome and fragile X-associated disorders. Coffee B. MosaicFMR1 deletion causes fragile X syndrome and can lead to molecular misdiagnosis: A case report and review of the literature. Garber K.

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Fragile X syndrome. Kidd S. Fragile X syndrome: A review of associated medical problems. Hagerman R. Hinton R. Parental reports on early language and motor milestones in fragile X syndrome with and without autism spectrum disorders. Cordeiro L. Autism: Current Theories and Evidence. The Humana Press Inc.

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Autism in genetic intellectual disability: Insights into idiopathic autism; pp. Smith L. Daily health symptoms of mothers of adolescents and adults with fragile X syndrome and mothers of adolescents and adults with autism spectrum disorder. Autism Dev.

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Abbeduto L. Psychological well-being and coping in mothers of youths with autism, down syndrome, or fragile X syndrome. Kronk R. Prevalence, nature, and correlates of sleep problems among children with fragile X syndrome based on a large scale parent survey.


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Gallagher A. Fragile X-associated disorders: A clinical overview.


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    A controlled study of longitudinal IQ changes in females and males with fragile X syndrome. Sansone S. Improving IQ measurement in intellectual disabilities using true deviation from population norms. Raspa M.

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    Public health literature review of fragile X syndrome. Advances in the treatment of fragile X syndrome. Lozano R. Fragile X syndrome: A review of clinical management. Intractable Rare Dis. Devys D. The FMR-1 protein is cytoplasmic, most abundant in neurons and appears normal in carriers of a fragile X premutation.