The orphan drug tax credit is crucial to assist and encourage pharmaceutical companies to develop therapies for rare diseases. Please help us spread the word to lawmakers by clicking on the button below. History An Orphan Drug is a pharmaceutical that is created in order to treat a rare disease. The orphan drug tax credit...
Community Conversation Recap: For Families to Discuss the Impacts of Dr. van Bosse leaving Shrinerโs Hospitals
On Tuesday evening, PWSA | USA hosted a Community Conversation bringing together members of the PWS community to informally share concerns, ideas, and updates on Dr. van Bosseโs recent departure from Shriners Hospitals. PWSA | USA CEO Paige Rivard has spoken directly with Dr. van Bosse who assured her that he remains committed to the...
PWSA | USA Family and Friends: We Need Your Help!
Attention PWSA | USA family and friends! We are working on a special project for our PWS community and are in need of pictures of you and your loved ones. We are creating a PWSA 2022 Calendar, which will be available in the next couple of months, and will include photo collages on each page....
Direct Support Professional Recognition Week
Direct Support Professional (DSP) is an umbrella term that includes many different titles and functions; for example: direct care, direct support worker/specialist/assistant/counselor, habilitation specialist, residential counselor, activities of daily living specialist, relief staff, apartment worker, developmental disabilities specialist, job coach, employment specialist, community bridge-builder, paid friend/neighbor, family care provider, family support services aide, community companion,...
FDA Advisory Committee to Review LEVO’s Carbetocin as a Treatment for PWS
We are excited to share that Levoโs New Drug Application (NDA) for carbetocin as a treatment for PWS will be discussed at a public meeting of the Psychopharmacologic Drugs Advisory Committee to be held November 4, 2021. This is a major step forward on this drug's path through the approval process. The Food and Drug...
Community Conversation Opportunity: For Families to Discuss the Impacts of Dr. van Bosse leaving Shrinerโs Hospitals
DOWNLOAD COMMUNITY CONVERSATION FLYER
Soleno Therapeutics Announces Positive Data Showing Continued Significant Improvements in Symptoms of PWS following One Year Treatment with DCCR
(Soleno Therapeutics Press Release) Statistically significant reduction in hyperphagia and all other PWS behavioral parameters in Study C602 Statistically significant improvements compared to natural history of PWS from the PATH for PWS Study On track for data submission to the FDA in Q3 2021 REDWOOD CITY, Calif., Sept. 08, 2021 (GLOBE NEWSWIRE) -- Soleno Therapeutics,...
NEW Customizable PWS Health Identification Card!
To help our communities better understand Prader-Willi syndrome, how it affects those diagnosed, and what symptoms require immediate medical attention, PWSA | USA has created a customizable PWS Health Identification Card. This Health ID card can be printed and shared with anyone in your community, e.g. school officials, medical providers, family members, etc. Download the...
Creating Your Childโs Special Education File
Creating a master file that contains the enormous amount of paperwork your child with special education needs will not only keep you organized and aid you in preparing for all interactions with the school district but will give you a clearer understanding of your childโs special education needs and progress. Schools often keep records in...
Understanding Gastric Motility and Gastroparesis in PWS
Compiled and Reviewed by Barb Dorn, RN, BSN, Margaret Burns, RD, Prader-Willi Homes, and Dr. Ann Scheimann, MD What is Gastric Motility and Gastroparesis? GASTRIC MOTILITY is the rate at which the stomach empties. GASTROPARESIS is slow emptying of the stomach.ย This condition occurs when there is a delay or slowing in the contraction of...
A Message from PWSA | USA’s CEO Paige Rivard Regarding Dr. van Bosse
A message from PWSA | USAโs CEO Paige Rivard: As many of you have heard, Dr. Harold van Bosse is no longer with Shriners Hospitals for Children in Philadelphia. I was in contact with Dr. van Bosse who said he is in the process of moving his practice and is still very committed to the...
We Are Brave Together
Founded in 2017 by Jessica Patay, โmother to a 18-year-old son with Prader-Willi syndrome, We Are Brave Together (WABT) is a nonprofit organization whose mission is to support caregiving mothers to children, any age, with any diagnosis, disability, or challenge, including learning differences, ADHD, and mental health issues. This international community includes over 1350 moms...
Show Your Support for the STAT Act!
Make your voice heardโฆSTAT! The EveryLife Foundation for Rare Diseases is seeking advocates to share quotes, photos, and/or videos to express their support theย Speeding Therapy Access Today Act, or STAT Act. Advocates are encouraged to share how their specific rare disease journey will be positively impacted by a specific aspect of the STAT Act. Please...
Donor Spotlight: Huma Onorato
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 will spotlight a donor and their story showcasing...
Webinar Opportunity: Become an Advocate for PWSA | USA
We are excited to offer a special webinar Thursday, August 26, 2021 at 8:00 p.m. EST on becoming an advocate for PWSA | USA. Advocacy is not just for lobbyists: Anybody can be an effective advocate. Join PWSA | USA and learn how you can help bring PWS awareness to states throughout the country. At...
The BENEFIT Act Receives New Co-Sponsor in U.S. House of Representatives
The BENEFIT Act, HR 4472 / S373, has obtained more bi-partisan support in the US House of Representatives! Representative Doug LaMalfa (R-CA) has signed on to show his continued support for the rare disease community. The BENEFIT Act is a bi-partisan piece of legislation that would require that the FDA disclose whether and how patient...
Harmony Biosciences Acquires Asset with Novel Mechanism of Action for the Potential Treatment of Narcolepsy and other Rare Neurological Diseases
Harmony Biosciences recently announced the acquisition of HBS-102, a potential first-in-class molecule with a novel mechanism of action, from ConSynance Therapeutics, Inc. HBS-102 is a Melanin Concentrating Hormone Receptor 1 (MCHR1) antagonist that has the potential to offer a novel approach to the treatment of narcolepsy including the symptoms of Rapid Eye Movement (REM) sleep...
WATCH: Homeschooling Webinar
If you didn't get a chance to join Monday's Homeschooling Webinar, the recording is now available below. Special thank you to Julie Casey and Danielle Warmuth for sharing helpful information about curriculums, services, and socialization!
The BENEFIT Act Receives Bill Number in U.S. House of Representatives
The BENEFIT Act, HR 4472 / S373, has received a bill number in the US House of Representatives! Representative Doris Matsui (D-CA) introduced the BENEFIT Act mid-July, but with a bill number itโs now easier than ever to ask your elected officials to sign on and support this life-changing piece of legislation. The BENEFIT Act...
August is Gastroparesis Awareness Month
August is Gastroparesis Awareness Month. Gastroparesis, also known as delayed gastric emptying, is a chronic condition that affects the stomach muscles and prevents proper movement of food from the stomach to the small intestine.Nearly every person with PWS has some degree of slow gastric emptying. This month, in honor of gastroparesis awareness, we are asking...
The Stat Act Receives More Bi-Partisan Support
The STAT Act, HR 1730, just obtained more bi-partisan support in the US House of Representatives. Representative David Kustoff (R-TN) and Representative Josh Gottheimer, (D-NJ) have joined forces with 19 other US representatives to show their continued support for the rare disease community. The STAT Act will allow patients with a rare disease speedy access...
Webinar Opportunity | Homeschool: What You Should Know
We are excited to offer a special webinar Monday, August 9, 2021 at 7:30 p.m. EST on homeschooling and PWS. The COVID-19 pandemic forced parents to make difficult decisions regarding their childrenโs education.ย Join Julie Casey and Danielle Warmuth to learn from veteran homeschoolers about curriculums, services, and socialization. REGISTER IN ADVANCE HERE __________________________________________________________________________________________________________________ ...
Harmony Biosciences adds Cincinnati, OH site to its ๏ปฟPhase 2 Clinical Trial
Harmony Biosciences is currently in its Phase 2 clinical trial of Pitolisant, studying the safety and impact of an investigational medicine for excessive daytime sleepiness, cognition, and behavioral function in people with PWS. This week, they added an 11th location to their list of trial sites around the U.S. The trial site will be located...
One Voice, One Message, One Goal
If you are interested in spreading awareness of PWS, engaging elected officials, and traveling with fellow PWSA | USA supporters, YOUR time has come! PWSA | USA is looking for volunteers from each state to participate in launching a special project to promote PWS awareness throughout the United States. For more information, please contact Dorothea...