Can't stop, won't stop! That's how we roll! It is 2022 and we are continuing our campaign to raise critical research dollars for Prader-Willi syndrome (PWS) research, and we need YOUR help!
Evan is 16 and in 10th grade. We are feeling a bit like experts at how to live this PWS life, but we know it could be better. We don’t want Evan to just get by…we want him to live his BEST life!
In June, we were given some disappointing news that a clinical trial drug that Evan had been taking for 3 years was being discontinued because the FDA declined to approve it. This has meant a drastic change has occurred in Evan's quality of life and the hyperphagia and anxiousness that had been well managed while on the drug are back and at an all-time high. We are saddened that this drug has been taken away but remain hopeful because this has shown there are treatments that make significant improvements in the most challenging aspects of Prader-Willi syndrome. We know we will need an arsenal filled with options to individualize the treatment for the best chance of success and more work needs to be done!
PWS has a constellation of symptoms, some of which are life limiting like severe behavior and temper outbursts that prevent individuals with PWS from living a full life. That is why we need your help. With your support, and careful stewardship, your donation dollars have powered past projects like the first-of-its-kind PWS Genome Project looking for variants that explain the characteristics of PWS and possible targets for future treatment and provided the seed money for the initial research supporting the development of the clinical trial drug DCCR for hyperphagia treatment. In 2022, it will power FPWR's largest project to date using tVNS (transcutaneous Vagus Nerve Stimulator) technology in a multi-center large study aimed at improving behavior and temper outbursts. This technology has shown promising results in pilot studies and has dramatically improved the lives of the study participants. Donations will also power other clinical projects aimed at improving daily life, genetic research and development of basic science projects so we can understand what is happing in the body that causes the issues faced by people with PWS and understanding the root cause is the first step to finding a treatment.
We remain humbled and grateful to all those who have donated in the past and who donate again this year. Your support of our family and all those with PWS is incredibly generous! In a time when there are so many causes to support, we are thankful you choose ours!
You can learn more about PWS and the research that is underway at http://www.fpwr.org/.