According to the National Institutes for Health, there are currently 20 investigational studies (either ongoing or preparing to enroll participants) that are specifically designed for Prader-Willi Syndrome. These studies are spread throughout the world, in places like the US, Canada, France and Taiwan. The topics being investigated include EDS (excessive daytime sleepiness), hyperphagia, behavior moderation, the gut microbiome and others.

These are exciting and promising times! So many ideas are being generated and so much potential is available. We have many world class physicians and researchers focusing their considerable talents on the myriad of symptoms and difficulties contained within the diagnosis of PWS. In my opinion, none more so than Dr. Jennifer Miller at the University of Florida.

As I’m sure most of you know by now (I believe I may have mentioned it a time or 2), Dr. Miller is Clem’s PWS specialist. Since birth, Clem has benefited from Dr. Miller’s expertise and willingness to think outside of the box. Clem has had the opportunity to try medications that are experimental. I believe these medications have softened some of the symptoms that Clem experiences.

But there is SO MUCH MORE to do. Hyperphagia has no current treatment. That symptom alone will prevent Clem from being able to live independently, even if all of the other symptoms are resolved! Mental health still presents serious challenges and concerns and research in this area is sorely lacking. Advances in understanding behavioral issues are slowly progressing but the pace must be quickened.

Advances in research will hold the key to improving the lives of those living with PWS. Without research there will be no answers or accumulation of understanding of this most complex and difficult condition. This is not something I’m willing to accept!

The simple fact is that research only exists when there are the financial resources to feed its appetite. I have committed myself to finding the resources to fund PWS research at the University of Florida. I have been very fortunate to find so many other amazing and dedicated families that share my goal. Together we will continue to raise money and generate hope for the future. There is no other path forward.

Nothing in life is to be feared, it is only to be understood. Now is the time to understand more, so that we may fear less.

Marie Curie
Clementine at school today. She is channeling Elton John and Jojo Siwa at the same time!