April 10, 2022 is National Siblings Day, a day where we take the opportunity to celebrate and recognize the amazing siblings in our PWS community. One sibling in particular is going above and beyond to raise awareness and funds for our rare disease community. Joe Gill, 22, will be running in the upcoming Boston Marathon...
FDA Advocacy in Action Webinar Registration Now Available!
Advocacy in Action presents… Engaging with the FDA: Opportunities and Boundaries WHEN: April 27, 2022 at 3:00PM EST REGISTER HERE As our community continues down the ever-evolving road of drug trials to enhance the lives of those who have Prader-Willi syndrome (PWS), PWSA | USA invites you to join us for a live webinar featuring two...
Clinical Trial Sites Announced for Radius Health’s RAD011 SCOUT-015 Study
Radius Health has announced the locations for its RAD011 SCOUT-015 Clinical Trials. Below, you will find a downloadable map with each participating hospital and medical center around the country. The RAD011 SCOUT-015 studies are in various stages, depending on the location, from initiated to activated and ready to enroll. You can also find a comprehensive...
April Advocacy in Action Webinar will Feature Guest Speakers from the FDA
SAVE THE DATE – APRIL 27, 2022 Time: TBD Registration Link Coming Soon! Advocacy in Action presents… Engaging with the FDA: Opportunities and Boundaries As our community continues down the ever-evolving road of drug trials to enhance the lives of those who have PWS, PWSA | USA would like you to join us as we bring...
Saniona Pausing All Clinical Trials for PWS Drug Tesomet Due to Funding Limitations
This morning, Saniona announced they will be voluntarily pausing all Phase 2b Clinical Trials for the drug Tesomet, which is being studied as a treatment for Prader-Willi syndrome (PWS). Saniona has explained that this pause is due to funding limitations and has nothing to do with the "safety or efficacy" of the drug itself. PWSA...
Lawmakers Review Several Legislative Proposals Intended to Accelerate Innovation of Therapies, Including STAT Act
On Thursday, March 17th, the House Energy & Commerce Subcommittee on Health held a hearing regarding The Future of Medicine: Legislation to Encourage Innovation and Improve Oversight. This hearing explored a number of legislative proposals intended to accelerate innovation of therapies, while ensuring appropriate levels of oversight, including H.R. 1730, the Speeding Therapy Access Today...
Texas Prader-Willi Association Opens New PWS Group Home
While the world and our Prader-Willi syndrome (PWS) community have been dealing with the pandemic's unpredictable circumstances, specifically the many obstacles to find appropriate supported living, the Texas Prader-Willi Association has managed to open a new PWS specific group home. The Texas Prader-Willi Association purchased and renovated a three-bedroom house to serve as a PWS...
What Type of Research Matters to You?
For more than 40 years, PWSA | USA has played a critical role in sponsoring and advancing research for the benefit of our PWS community. We are excited to continue this important commitment to PWS research in ways you have always relied on (organizing scientific conferences, offering grants to clinicians, etc.). We are also eager...
STAT Act to be Discussed in Congressional Hearing – Get in Touch with your Members of Congress!
URGENT CALL TO ACTION!! Due to the tremendous momentum created by Rare Disease Week on Capitol Hill, The Speeding Therapy Access Today (STAT) Act will FINALLY be discussed in a Congressional hearing on Thursday, March 17th at 10:30AM EST! This is a MAJOR milestone across all rare disease communities' efforts to pass targeted and impactful...
PWSA | USA’s 2021 Annual Report
Dear friends, On behalf of PWSA | USA's Board of Directors and Staff, we are sincerely grateful for YOU, our PWS community, for coming together in advocacy, family support, and research over the past year. We would not be able to offer the resources, care, and hope to our families and individuals living with PWS...
April Webinar Series to Provide Health Care Advocacy Tools, Resources for PWS Community
In April 2022, PWSA | USA will host the "Find Your Voice: Advocating for PWS Health Care" webinar series to provide helpful tools and resources on health care advocacy for our PWS community. Because there are very few health care professionals in our communities who have knowledge of the unique health issues of infants, children,...
Thank You for Shining a Light on Rare Disease Day!
Thank you to our PWS community for helping us shine a light on Rare Disease Day! This year, the National Organization for Rare Disorders (NORD) focused on encouraging buildings, landmarks, and monuments around the world to participate in a chain of lights to recognize Rare Disease Day. We are excited to share that many buildings/landmarks/monuments...
Help PWS experts learn more about feeding tube use in PWS
The information below was provided by the Global PWS Registry --------------------------------------------------------------------------------------- We know feeding tubes are often used in infancy for our loved ones with PWS who have difficulty feeding in the early months after birth. If your child used a feeding tube, we are asking you to spend 10 minutes today completing the new...
Global PWS Registry Shares Latest Orthopedic Data for Individuals Living with PWS
The information below was provided by the Global PWS Registry and approved by the Institutional Review Board (IRB) -------------------------------------------------------------------------------------------- Physical activity and exercise are an important part of care for individuals with PWS. However, this can be difficult due to poor muscle tone and orthopedic issues. Many individuals require bracing, casting, and/or surgery for spinal issues....
Thank you for Sharing your Rare!
Thank you to those who shared their rare to help shine a light on Rare Disease Day!
My Brother Daniel
Written by Daniel's Sister _________________________________________________________________________________ My brother, Daniel, was born in 1956, the same year that PWS was formally identified but he was not diagnosed until he was sixteen. Our family, (two parents, Daniel, and his four sisters) moved around a lot — from the U.S. to Chile, France, Canada and Venezuela—eventually settling in the...
PWSA | USA Volunteer Appreciation Award Nominations Now Being Accepted!
Help us honor our PWSA | USA volunteers! Do you know someone who deserves to be recognized for their service and dedication to PWSA | USA's mission and the PWS community? 2022 Volunteer Appreciation Award nominations are now being accepted through March 15, 2022. PWSA | USA will give out the following four awards: Advocacy...
PWSA | USA CEO Paige Rivard Participating in Webinar Alongside U.S. Representatives Ahead of Rare Disease Day
As the nation gets ready for Rare Disease Day on February 28, 2022, PWSA | USA CEO Paige Rivard will participate in a national webinar hosted by Harmony Biosciences on Thursday, February 24th at 1pm EST to discuss COVID-19 and Rare Disease Patients. Paige will join U.S. Representative Josh Gottheimer (D-NJ), U.S. Representative Brad Wenstrup...
Prader-Willi Association of New England’s Trivia Night
Mike “Sarge” Riley, Professional Sports Announcer and Boston Media Personality from 98.5 will host a virtual trivia night fundraiser for the New England Chapter of the Prader Willi Syndrome Association WHEN: Friday March 11 at 7:30 pm (about an hour) WHERE: Online-via Zoom You will receive a zoom link and a reminder before the event....
PWSA | USA’s Family Support Team Recaps January, February Community Conversations
January Community Conversation: Guardianship Lisa Thornton is not only a parent of a young woman with PWS, but she is an attorney with a lot of experience navigating guardianship and special needs trusts. If, when, and how to apply for guardianship is often a daunting process for parents. Coupled with every state having different processes,...
Apply to Attend PWSA | USA’s 1st Annual Volunteer Summit!
APPLICATION DEADLINE: Friday, March 4, 2022 Our PWSA | USA Volunteer Summit is a unique opportunity to unite, support, and celebrate PWSA | USA volunteers who share a passion for supporting individuals and families within the PWS community. In addition to celebrating our volunteers, staff and special speakers will share new information, host small group...
Join us for the March Advocacy in Action Webinar with Speaker Maureen Tracey
When: March 9, 2022 at 8:00 p.m. EST Topic: Navigating and Advocating the Special Education Process for Students with PWS REGISTER IN ADVANCE HERE Advocacy In Action is happy to welcome one of our own as our guest presenter for the month of March: Maureen Tracey, Founder and CEO of Four Seasons Behavioral Solutions As...
Brain Tissue Donation: Honoring Loved One’s Through Hope
Autism BrainNet is a program of the Simons Foundation Autism Research Initiative (SFARI) that promotes innovative, high-quality research on postmortem brain tissue to improve the understanding of the biological causes of autism spectrum disorder (ASD) and related neurodevelopmental conditions. We work with Autism BrainNet to promote a greater knowledge of neurodevelopmental conditions and brain tissue research,...
Learn More About Harmony Biosciences Phase 2 Study of Pitolisant for PWS
Does your loved one struggle with daytime sleepiness? Would you like to learn more about a potential treatment for daytime sleepiness, cognition, and behavior? Join PWSA | USA, FPWR, and Harmony Biosciences Wednesday, February 16th at 3:30 pm ET for a short presentation followed by Q&A to learn more about the Phase 2 study of...