Summary of the Impact of PWS on Individuals and Their Families and Views on Treatments: Results of an International Online Survey


PWS is a life threatening disorder, with lifelong consequences for the person with PWS and their family. PWS presents with a myriad of medical issues, and is characterized by a challenging behavioral and mental health phenotype. The inability to control food intake represents the major impediment to independent living for individuals with PWS, who must be supervised 24/7 with highly restricted access to food. Thus, they are unable to work in environments where food is accessible (i.e, almost all work environments), nor shop for or prepare food independently. Social interactions and opportunities for community engagement and are highly constrained by food access issues, isolating the individual and their family, which likely contributes to the chronic stress and the mood/health impacts that families experience in the long term.

Few therapies are available for those with PWS. Growth hormone (GH) therapy was FDA approved in 2000 for children with PWS and increasingly prescribed in infants and adults as well. GH therapy improves growth, body composition, endurance and development, but does not impact hyperphagia. Presently, there are no drugs that improve hyperphagia in PWS, and only limited therapeutic options for mitigating behavioral and psychiatric challenges. However, a number of potential therapies are in development. To better guide the development of drugs that are meaningful to individuals with PWS and their families, it is critical to understand the severity and impact of symptoms associated with PWS, as well as the usefulness of current therapies and the characteristics of an ideal therapy. The PWS ‘Patient Voices Survey’ was developed by patient advocacy groups and conducted as a first step in ascertaining the views of families who have a child with PWS as it relates to symptoms, unmet medical needs, drug development priorities and attitudes towards clinical trials. An online “Patient Voices” survey was distributed to the PWS community through social media, with 779 responses gathered over a two-week period in Oct-Nov 2014.


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Patient Voices Webinar, November 2014

A webinar was presented in November os 2014 summarizing data gathered from the PWS community. Here are slides from that webinar: Patient Voices Webinar Slides. Click the video below to watch.