Category: News

Empowering Diversity and Inclusion for the PWS Community

In a significant step towards fostering equity and inclusion within our Prader-Willi syndrome (PWS) community, we are pleased to announce a collaborative initiative aimed at improving the standard of care and support for marginalized individuals living with PWS and their families. This initiative, undertaken with the active involvement of several community members, seeks to ensure...

Fueling Hope: Aardvark Therapeutics’ $85M Boost Powers Breakthrough PWS Treatment

Aardvark Therapeutics has just hit a major milestone with an exciting $85 million Series C financing round! Led by Decheng Capital, with additional participation from several investors, including PWSA | USA, this funding is set to drive the development of ARD-101, a groundbreaking treatment for hyperphagia in Prader-Willi syndrome patients. ARD-101 showed promising early trial...

Rare Disease Advocates Will Unite in Washington, D.C., to Celebrate National Prader-Willi Syndrome (PWS) Awareness Month, National PWS Awareness Day

May 8, 2024 (Washington, D.C.) — Prepare for a powerful convergence in the heart of Washington, D.C., on May 15th as advocates for individuals living with Prader-Willi syndrome (PWS) gather for PWSA | USA’s important advocacy event, the D.C. Fly-In. This year marks the 2nd National PWS Awareness Day, a significant milestone achieved through the...

Preserving Progress: Stand Up for Fully Funding the BRAIN Initiative!

The NIH-BRAIN Initiative (Brain Research Through Advancing Innovative Neurotechnologies – Initiative), is a partnership between the National Institutes of Health (NIH) and nonfederal funding sources to advance innovative neurotechnologies to revolutionize the understanding of the brain/neurocircuitry. Since its inception in 2013, this program has had important impacts on our understanding of normal brain function this...

Prader-Willi Syndrome Research

Request for Prader-Willi Syndrome Research and Mini-Fellowship Grant Applications

PWSA | USA is currently seeking research project applications with direct impacts on individuals and families affected by PWS. We are offering mini-fellowship grants to support providers in enhancing their understanding of PWS through clinical proctorships. Research priorities include expanding knowledge about PWS, applying therapies, and attracting new providers and investigators to the field. Funding...

Breaking Ground: FDA Grants Breakthrough Designation for PWS Drug Development

Big news! Soleno Therapeutics has announced a groundbreaking achievement: diazoxide choline (DCCR) has been granted Breakthrough Therapy Designation by the FDA for Prader-Willi syndrome (PWS). This marks a significant milestone as the FIRST-EVER designation for a drug developed for PWS. The designation underscores the FDA’s recognition of PWS as a serious condition and the potential...

Calling All PWS Community Members: Sign the FDA Petition

Attention everyone in the Prader-Willi syndrome (PWS) community and beyond! We need your support NOW! We are rallying behind a critical petition urging the FDA to take action on DCCR (diazoxide choline) for individuals living with PWS. Soleno Therapeutics’ investigational drug has shown remarkable promise in improving hyperphagia, reducing fat mass, and addressing challenging PWS-associated...

Harmony Biosciences Initiates TEMPO PWS Study

Harmony Biosciences Holdings, Inc. is seeking participants for its TEMPO study, a global Phase 3 trial investigating pitolisant as a potential treatment for excessive daytime sleepiness (EDS) in individuals aged six years and older with Prader-Willi syndrome (PWS). Pitolisant is a medication that could help manage sleepiness and behavioral issues in people with PWS. There...

Blue backgound with Harmony Bioscienes label for pitolisant as treatment for Prader-Willi Syndrome

Pitolisant Receives Orphan Drug Designation

Pitolisant from Harmony Biosciences Receives Orphan Drug Status from FDA for Treatment of PWS The FDA has granted Orphan Drug status to pitolisant, the trial drug from Harmony Biosciences to treat excessive daytime sleepiness (EDA) and behavioral disturbances. The designation shows that the FDA considers pitolisant to be a promising treatment for people with PWS. ...

PWSA USA Development Specialist Position

PWSA | USA Now Hiring for Director of Accounting, Development Specialist Positions

Position: Full Time Development SpecialistLocation: Remote Do you have a passion for cultivating relationships and supporting those affected by PWS? Here is your opportunity! PWSA | USA is on the lookout for a dynamic individual to join our growing development team! As the Development Specialist, you’ll play a crucial role in special projects, grassroots support,...

Map of States the recognize PWS as a Developmental Disability

Joint Legislation Filed to have PWS Recognized as a Developmental Disability in Ohio

PWSA | USA is excited to announce that thanks to the dedication and hard work of our Advocacy Ambassador, Dr. Erin Carter-Cooper, PhD, Ohio Representative Rose Sweeney (D - District 16) and Representative Gayle Mannin (R – District 52) have jointly filed legislation that will make Prader-Willi syndrome a recognized developmental disability in the State of Ohio!...

PWSA | USA Announced as one of Harmony Biosciences 2023 Patients at the Heart Recipients

PWSA | USA Announced as Harmony Biosciences’ 2023 Patients at the Heart Grant Recipient

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...

PWSA USA Advocates to Participate in U.S. Senate Committee Hearing

PWSA | USA Advocates will Participate in Groundbreaking Opportunity for PWS Community!

U.S. Senate Committee Hearing Live Broadcast Link Now Available This Thursday, PWSA | USA advocates will participate in a groundbreaking opportunity for the PWS community! On October 26, 2023, our advocates will be present for a press conference on Capitol Hill, hosted by Senator Mike Braun (R-IN), Senator Kirsten Gillibrand (D-NY), Representative Mike Gallagher (R-WI),...

Soleno Therapeutics Reports Positive Results from DCCR Study C602 for Prader-Willi Syndrome

Soleno Therapeutics, Inc. has revealed positive outcomes from the randomized withdrawal phase of Study C602, an extended treatment study of DCCR (Diazoxide Choline) Extended-Release tablets for Prader-Willi syndrome (PWS). The results, which showed a significant improvement in hyperphagia-related behaviors in the DCCR group compared to the placebo group, support Soleno's plan to submit a New...

Harmony Biosciences Issues Statement Regarding Confidence in Pitolisant Drug

Harmony Biosciences has reaffirmed its confidence in the strength of WAKIX® (Pitolisant) patents, after receiving a positive ruling from the U.S. Patent and Trademark Office (USPTO) rejecting the request for reexamination. WAKIX® is used to treat excessive daytime sleepiness (EDS) or cataplexy in adults with Narcolepsy. Read Harmony Biosciences' community-facing statement below: We are pleased...

Harmony Biosciences Shares Positive Data from Pitolisant Study at “SLEEP 2023” Annual Meeting

PWSA | USA received information from Harmony Biosciences that the company announced positive findings from the Phase 2 study of its drug pitolisant, a treatment for excessive daytime sleepiness (EDS) in people with Prader-Willi syndrome (PWS). The data was presented at the 37th Annual Meeting of the Associated Professional Sleep Societies (APSS). According to Harmony...

A Historic Milestone for the PWS Community: PWSA | USA Receives Joint Congressional Support to Declare May 15th as National PWS Awareness Day

Today, May 15th, our PWS community has something new to celebrate! PWSA | USA is excited to announce U.S. Congressman Paul D. Tonko (D-NY) and Congresswoman Maria Elvira Salazar (R-FL) have filed a joint resolution declaring May 15, 2023, and every May 15th thereafter, Prader-Willi Syndrome (PWS) Awareness Day in the United States of America!...

Harmony Biosciences Shares Statement Regarding Narcolepsy Drug WAKIX

In response to yesterday’s news from Scorpion Capital regarding Harmony Biosciences, we have received the below community-facing statement. Harmony Biosciences shares information about the company’s drug WAKIX, which is used to treat excessive daytime sleepiness (EDS) or cataplexy in adults with Narcolepsy. If you have questions, please reach out to info@pwsausa.org. —————————————————————————- On behalf of...

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