DCCR Townhall Meeting

Hyperphagia is surely the most infamous of the PWS symptoms. Back in those dark early days when we received Robyn’s diagnosis I couldn’t focus on much else. Like most of the population I’d never heard of hyperphagia and was shocked that such a cruel symptom could exist. How could anyone live with the feeling of constant hunger? My daughter is only two years old and as such it still feels somewhat abstract but none the less, it feels to varying extents, like an ever present cloud on the horizon.

Hyperphagia is a debilitating symptom of PWS that causes those with PWS, caregivers and families significant anguish. This is one of the reasons why research is so important and why drugs such as Soleno’s DCCR are so desperately needed.  Like many others in the PWS community I was devastated when I heard that the FDA in America had asked for more data by way of a further trial.

Thankfully the PWS community has contested this decision with FPWR and PWSA USA teaming up to form a unified voice. To this end a virtual townhall gathering was recently held to show some very moving testimonies from families and individuals with PWS. Those testimonies highlighted how DCCR has positively impacted their lives. It was amazing and filled me with hope! For some the impact of the drug has been smaller (though still significant) whilst for others completely transformational. Interestingly the drug seems to have positively impacted other aspects of PWS such as reducing anxiety and increasing muscle tone.

Unfortunately it seems Covid negatively impacted the study and skewed the final data in the study. The PWS community has requested that the FDA use discretionary flexibility and waive the need for a challenging and lengthy additional trial. Having watched the townhall meeting, there are certainly many compelling reasons to hope and indeed believe that this request prevails.

One thing for sure is we are part of a resilient and determined community and that fills me with great hope for the future.

This blog was written by David Jack, father to Robyn who has PWS. They live in the Scottish Highlands.

You can access the townhall meeting by clicking on this link