Foundation for Prader-Willi Blog | Research

Several NEW PWS trial opportunities are now available. FPWR maintains the latest clinical trial information so that you can stay abreast of trial opportunities. Some trials require in-person visits while others can be completed remotely.  Some trials are testing new drugs while others are intervention (non-drug) or natural history studies, like the PWS Weight Study described below. 

FPWR does not endorse or recommend any specific study. Our goal is to provide information to help members of the PWS community decide whether participation in a clinical trial is right for them and their loved one with PWS.  Anyone considering participating in a clinical trial should discuss the matter with his or her physician. 

The results of a Phase 2 clinical trial evaluating carbetocin in PWS have just been published in the journal JCI Insight. The findings of the paper, "Intranasal Carbetocin Reduces Hyperphagia In Individuals With Prader-Willi Syndrome," support the advancement of the drug to a larger and longer Phase 3 evaluation in the PWS population.

On May 15, PWSA-USA and FPWR jointly presented the webinar How Patients Can Partner to Speed Treatments for PWS. The webinar provided important information about PWS clinical trials including: what to expect when participating in a trial, what questions you should ask, and how to get the information you need to decide if a clinical trial is right for your loved one with PWS. The webinar also gave a brief overview of the trial opportunities that are available in 2018. You can watch the webinar in its entirety or read a summary of the webinar below.

FPWR congratulates Dr. Elisabeth Dykens, who was awarded last night with a Rare Impact Award by the National Organization for Rare Disorders.

Dr. Dykens may best be known in the Prader-Willi syndrome (PWS) community for developing the Dykens Hyperphagia Questionnaire, an assessment of the food-seeking behaviors common among individuals with Prader-Willi syndrome, which has become the gold standard for measuring outcomes in PWS clinical trials.

Soleno has announced the opening of their first clinical trial site for a Phase 3 study of DCCR to measure the drug's efficacy for treating hyperphagia in PWS. That site is Seattle Children’s Hospital.

The Foundation for Prader-Willi Research announces our first round of Research Awards in 2018 totaling $615,000. FPWR is dedicated to supporting research that advances the understanding and treatment of Prader-Willi syndrome (PWS) and to that end, has awarded over $10,000,000 to research since 2003.

Anxiety is a common challenge for our loved ones with PWS. Recently, we reviewed responses in the Global PWS Registry related to anxiety. According to registry respondents, anxiety is an issue for 48% of individual with PWS ages 10 and up.

20 unique paintings are available to the highest bidders in a unique art auction benefiting the Foundation for Prader-Willi Research (FPWR).

It is an exciting time in PWS research, with multiple new clinical trials on the horizon this spring and through the end of 2018! There is already buzz throughout the community about upcoming studies, providing a wonderful opportunity for an open dialogue about the clinical trial process. FPWR does not advocate certain trials over others. We aim to provide resources for information, so that families are empowered with the knowledge and education to make the best decisions for themselves.

FPWR is partnering with Autism BrainNet to collect and study post-mortem brain donations. Through  this partnership, we aim to raise awareness about the importance of post-mortem brain donation, streamline the donation process for families, and enhance the collection and distribution of high-quality tissue to researchers.