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...
Category: Blog
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!
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...
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,...
Be PWSA | USA’s Valentine!
Share Your Heart with PWSA | USA on Valentine's Day Forget the flowers! Cancel the chocolate! Please be OUR Valentine with your gift to PWSA | USA's Hearts of Hope campaign between Tuesday, February 8, 2022 and Sunday, February 20, 2022. By making a donation to the Hearts of Hope campaign, you will have the...
February Donor Spotlight
PWSA | USA is special because of YOU, our donors, who passionately give to support our mission. It is because of your generous gifts that we can provide help and hope through family support, advocacy, and research efforts. To celebrate and honor your generosity, each month we spotlight a donor and their story showcasing the...
Harmony Biosciences Now Enrolling Individuals with PWS for Upcoming Clinical Trial
Harmony Biosciences has released information about their PWS Phase 2 clinical trial where they will be studying "the safety and impact of an investigational medication on excessive daytime sleepiness, cognition, and behavioral function in people with PWS." Harmony Biosciences is currently looking for individuals with PWS between the ages of 6-65 to enroll in this...
Clint Hurdle Hot Stove Dinner: A Look Back at the Past, Forward to the Future
As we get ready for the 8th Annual Hot Stove Dinner, which will be held on March 26th at the Key Royale Club on Anna Maria Island, Florida AND online for virtual guests, event host Clint Hurdle takes us on a trip down memory lane! Below, Clint shares how he and his wife Karla created...
Radius Health Shares Information about its Drug RAD011, Upcoming Study in New Fact Sheet
January 24, 2021 -- Radius Health, Inc. recently unveiled a detailed fact sheet about its drug RAD011, a synthetic cannabidiol oral solution, which is being studied as a possible treatment for hyperphagia and related behaviors in Prader-Willi syndrome. The fact sheet also gives a description of Radius Health's upcoming SCOUT-015 clinical trial. According to Radius...
Soleno Therapeutics Provides Update on DCCR
January 24 ,2022 -- PWSA| USA is sharing the most recent news released on the status of Soleno Therapeutics' drug DCCR (diazoxide choline) for the treatment of Prader-Willi syndrome. Soleno announced today that it has received the official minutes from a December 2021 meeting with the FDA's Division of Psychiatry. This meeting was a follow...
Join us for the February Advocacy in Action Webinar with Guest Speaker Guy Stephens
Advocacy In Action is excited to welcome our guest presenter for the month of February, Guy Stephens, Founder and Executive Director for the Alliance Against Seclusion and Restraint (ASSR) Join us February 9, 2022 at 8:00 p.m. EST for our next Advocacy in Action webinar, as part of our continuous series to raise awareness and...
Levo Therapeutics Receives Complete Response from FDA for its Decision on Carbetocin
January 18, 2022 08:00 ET | Source: Levo Therapeutics, Inc. CHICAGO, Jan. 18, 2022 (GLOBE NEWSWIRE) -- Levo Therapeutics, Inc., a biotechnology company dedicated to using genetic insights to advance treatments for Prader-Willi syndrome (PWS) and related disorders, announced today that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA)...