作者
Lauren Schwartz, Assumpta Caixàs, Anastasia Dimitropoulos, Elisabeth Dykens, Jessica Duis, Stewart Einfeld, Louise Gallagher, Anthony Holland, Lauren Rice, Elizabeth Roof, Parisa Salehi, Theresa Strong, Bonnie Taylor, Kate Woodcock
发表日期
2021/12
来源
Journal of Neurodevelopmental Disorders
卷号
13
页码范围
1-13
出版商
BioMed Central
简介
Prader-Willi syndrome (PWS) is a rare neurodevelopmental genetic disorder associated with a characteristic behavioral phenotype that includes severe hyperphagia and a variety of other behavioral challenges such as temper outbursts and anxiety. These behaviors have a significant and dramatic impact on the daily functioning and quality of life for the person with PWS and their families. To date, effective therapies addressing these behavioral challenges have proven elusive, but several potential treatments are on the horizon. However, a limiting factor for treatment studies in PWS is the lack of consensus in the field regarding how to best define and measure the complex and interrelated behavioral features of this syndrome. The International PWS Clinical Trials Consortium (PWS-CTC, www.pwsctc.org ) includes expert PWS scientists, clinicians, and patient advocacy …
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