FPWR Blog

An Update to the Prader-Willi community from Harmony Biosciences:

As you may know, the team at Harmony has been working toward initiating a Phase 2 clinical trial to assess the safety, efficacy and appropriate dosing of pitolisant in treating some of the symptoms experienced by patients with Prader-Willi syndrome.

A special contribution by guest blogger Felicia DiMuccio

Growing up, I was never quite sure what I wanted to be, but I always knew I wanted to be a mom. Finding out I was pregnant with Siena was a dream come true, and I couldn't wait for her to enter the world. My pregnancy was normal, and all the testing I had done confirmed that I was carrying a healthy baby girl. All of that changed on September 11, 2012. 

Today we received disappointing news from Millendo in regards to their Phase 2 study of Livoletide in PWS (Zephyr).  Unfortunately, results from the trial do not show the significant improvements in hunger that we had all hoped to see and Millendo has discontinued the livoletide program for PWS.

In our latest podcast, Patrice Carroll, Director of PWS Services at Latham Centers in Cape Cod, shares how to how to help our loved ones with PWS through this unusual time of social distancing and isolation due to COVID-19. To listen, just click above.

COVID-19 has turned all of our lives upside down. These times can be particularly challenging for our loved ones with PWS who rely on routines and schedules. In this webinar, Elizabeth Roof, Senior Research Specialist at Vanderbilt Research Center, shares strategies to manage repetitive behaviors and questions, as well as tips to help motivate your loved one to participate in activities such as at home learning and exercise. Elizabeth has kindly shared the slides from her presentation. Download the slides here.

In our latest podcast, Lauren Schwartz-Roth, clinical psychologist and mom to a young adult with PWS, shares how to identify and treat serious mental health issues in our loved ones with PWS. To listen, just click above, or read our partial transcript below.

In our latest podcast, Lauren Schwartz-Roth, clinical psychologist and mom to a young adult with PWS, shares tips on how to stay calm and manage anxiety during COVID-19. To listen, just click above, or read below for a list of tips to start using today.

You won’t want to miss these speakers at the 2020 FPWR Family Conference this October in Chicago, Illinois.

Inspiration, hope, people who get it — that’s the 2020 FPWR Family Conference! You don’t want to miss joining like-minded game-changers with direct access to the people, knowledge, and inspiration you need to help your child.  

The FPWR Family Conference takes place October 1-3, in Chicago, IL. Here are the top 10 reasons you need to be there!

10. Chicago!

Nightlife, live music, culture, and great food are all at your convenience! Shop and explore the Millennium Park and the Chicago Riverwalk; go on a tour of the city or visit the nearby zoo for family fun and outdoor recreation. The options are endless!

Soleno therapeutics recently completed enrollment of their ongoing Phase III Clinical trial, DESTINY PWS, and is expecting to announce top-line data the first half of 2020. The study is evaluating once-daily Diazoxide Choline Controlled-Release (DCCR) tablets for patients with Prader Willi Syndrome (PWS).