Making Each Day a Better Tomorrow for Individuals Living with PWS.
Our Research Statement
Many of the world’s most renowned PWS researchers and clinicians are members of PWSA | USA’s scientific and clinical advisory boards. Together they dedicate thousands of hours yearly to enhance research, write educational materials, and provide consultation; nearly all the major breakthroughs in understanding and treating PWS have come from these pioneers and heroes. PWSA | USA has been supporting research since 1983 and remains committed to supporting future advancements and breakthroughs.
At PWSA | USA we work to integrate what we have learned about the needs of our families through our support programs — with research that we think will make an important and practical difference in the lives of those affected by PWS
Our Research Strategy
The current focus of PWSA | USA research is supporting projects that have the potential for immediate and high impact for the PWS community. Therefore, PWSA | USA will support research-based translational grants or those showing high promise for translating basic biomedical knowledge to clinical application. The goal is to fast-track better treatment for the syndrome.
Post-Mortem Tissue Donation
As research into the biology of Prader-Willi syndrome (PWS) has become more sophisticated, studies on the postmortem human brain have become an increasingly important tool for understanding the neuropathology of the syndrome.
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, while ensuring the smoothest donation process for families during a very difficult time.
Studying postmortem brain tissue can lead to a better understanding of the cellular and molecular changes associated with ASD and related neurodevelopmental conditions in the human brain, as well as identify targets for new and effective treatments.
Autism BrainNet makes the donation process as simple as possible for every family. Donor families are treated with respect and compassion and provided with ongoing support. These are the steps involved in the process of brain donation to Autism BrainNet:
1. Call Autism BrainNet at 877-333-0999 for immediate assistance (optimally within 24 hours of the death of a loved one). If death is near, you can call Autism BrainNet to make plans for donation. Autism BrainNet’s staff is available 24/7 and will walk your family through the donation process.
2. Autism BrainNet coordinates brain recovery and transportation to their nearest site.
3. An Autism BrainNet clinician follows up with the donor family to schedule a video meeting or home visit to learn more about the donor.
There is no cost to families, and brain donation does not interfere with autopsy or funeral plans. Autism BrainNet’s staff can be contacted anytime to answer questions or concerns about the
brain donation process.
Pre-registration is not required to become a donor with Autism BrainNet. However, you can download and print the Intent to Donate Postmortem Brain Tissue for Research form and an Autism BrainNet Brain Donor Card to include in your preplanning documents.
Families are welcome to subscribe to the Autism BrainNet’s newsletter to receive program and scientific updates. To sign up, visit: autismbrainnet.org/newsletter.
Please copy and paste from the suggested messaging below.
ECHO 4 PWS Health Care Provider Series
PWSA | USA is excited to announce the launch of our new Health Care Provider Project ECHO Series. We are proud to offer this opportunity to health care professionals who work with patients affected by Prader-Willi syndrome (PWS) and to be the first PWS organization to implement Project ECHO® in the United States.
Register for the Global PWS Registry
The Global PWS Registry is a powerful tool for the PWS community to advance understanding of PWS, areas of unmet need, standards of care, and new therapies. Data from the registry is shared back to the PWS community and is also used by researchers and scientists. By participating, families of those with PWS become part of the research team, helping uncover trends in causes and diagnosis as well as new directions in therapies and treatment.
Articles of Interest
Find several PWS Articles of Interest across several categories, including genetics, brain imaging, growth hormones, sensory development, and physical more.
Visit our Blog
Acadia Pharmaceuticals has Announced the Initiation of its Phase 3 Study for Nasal Carbetocin to Treat Hyperphagia
This week, Acadia Pharmaceuticals announced the initiation of its Phase 3 COMPASS PWS study! This study focuses on evaluating the efficacy and safety of carbetocin nasal spray (ACP-101) for treating hyperphagia in Prader-Willi syndrome. The COMPASS PWS trial is a 12-week, double-blind, randomized, placebo-controlled global Phase 3 trial, with approximately 170 participants aged five to […]
About Harmony Biosciences Patients at the Heart Grant Via Harmony Biosciences Press Release: Harmony Biosciences Holdings, Inc. (“Harmony”) (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, has selected the latest round of nonprofit organizations for its Patients at the Heart and Progress at the Heart […]
Exciting news for the Prader-Willi syndrome (PWS) community! Newly unveiled data from Harmony Biosciences’ phase 2 study reveals promising impacts of pitolisant (Wakix) on PWS patients with excessive daytime sleepiness (EDS). Harmony Biosciences plans to kick off its phase 3 TEMPO study later this year, aiming to dive deeper into the potential of this treatment […]