Prader-Willi Syndrome Association | USA

Big news! The FDA has accepted Soleno Therapeutics' new drug application (NDA) for DCCR, a drug aimed at treating hyperphagia in people with PWS who are 4 years and older. Even better, the FDA granted Priority Review, which means they see this drug as a potential game-changer for improving the lives of those with PWS. While this is just the first step in the FDA's decision-making process, it's a promising development toward making DCCR available for our loved ones. A final decision is expected by December 27, 2024. Soleno Therapeutics CEO, Dr. Anish Bhatnagar, expressed gratitude to the entire PWS community, including patients, caregivers, and advocacy groups, for their continued support. You can read Soleno's full press release announcement at https://lnkd.in/e2_SpC67.

We are thrilled to announce the upcoming launch of PWS Connect, a brand-new podcast from PWSA | USA, debuting Friday, August 30, 2024! PWS Connect will be your go-to source for the latest news, research, advocacy, and family support in the Prader-Willi syndrome community. This podcast brings together voices from across the PWS landscape, offering valuable insights and practical advice to empower and support those affected by PWS. * On August 30th, check our website, social media, and Special Edition Pulse email newsletter for information on how to listen. Episodes will air every Tuesday and include valuable content to inform, inspire, and empower. We invite you to join us on this new journey!

Exciting News! 📣 On August 2, 2024, the PWS Advocacy Coalition submitted a petition to the FDA for the priority review of Soleno Therapeutics' drug, DCCR. This petition was signed by 14,271 PWS community members! While we expect the FDA to acknowledge the submission, they won't comment publicly due to confidentiality rules. Stay tuned for updates on the DCCR NDA review and decision. Read more and find the petition document at https://lnkd.in/g_tU6Ckz.

Join PWSA | USA and the team from Acadia Pharmaceuticals on Tuesday, July 23 at 8:00 p.m. EST / 5:00 p.m. PST to learn more about the COMPASS PWS Study, which aims to investigate the drug Carbetocin (nasal spray) as a treatment for PWS, specifically to help address hyperphagia symptoms. Acadia representatives will give details about the drug and discuss their Phase 3 study openings, requirements, and more! They will also be available for a Q & A discussion for any questions you have. We invite you to sign up for this free webinar opportunity you won’t want to miss! Register TODAY at https://lnkd.in/gn39PVun.

Exciting Advocacy News! PWSA | USA has submitted our "Congressional Letter of Support" to the FDA with 27 signatures from US Congress members! This letter pushes for urgent development and approval of treatments for Prader-Willi syndrome. A huge THANK YOU to our advocates who shared this ask during the 2024 D.C. Fly-In and to the team leads who worked diligently to follow up with their members of Congress. Your dedication is making a difference. You can read the letter at https://lnkd.in/gVSEze4P. Together, we’re bringing hope and a better quality of life to those affected by PWS. Stay tuned for updates!

We were fortunate to have PWSA | USA's 2024 D.C. Fly-In event captured in a unique and authentic way. Please enjoy this mini-documentary of our time on Capitol Hill, where nearly 150 parents, caregivers, siblings, family members, and individuals living with PWS came together to advocate for our community's needs. We are sincerely grateful to those who stepped in front of the camera lens to share their story. Together, we are driving change and making a lasting impact for those affected by PWS. Thanks to Soleno Therapeutics, Inc. for sponsoring the production of these videos. Produced by Believe Limited: The Patient People.

Join us for the PWS - Aging Research and Health Update Webinar series! 🗓️ Part 1: July 30, 2024, 8 PM EST | Register: https://lnkd.in/gUEagWUt 🗓️ Part 2: August 6, 2024, 8 PM EST | Register: https://lnkd.in/gWmKsHrG Learn about aging in PWS, common health issues, and prevention strategies from experts Lynn Garrick, RN, MSN, and Barb Dorn, RN, BSN. 📌 Topics include: - Dental concerns - Choking - Constipation - Diabetes - Respiratory issues - Weight gain/obesity - Low bone density - Cardiac issues - Mobility challenges - Mental health #PWS #HealthWebinar #AgingResearch

📢 Exciting News from Soleno Therapeutics! 🎉Soleno announced today the company has officially submitted a New Drug Application (NDA) to the FDA for DCCR (diazoxide choline) extended-release tablets. This new treatment targets Prader-Willi syndrome (PWS) in individuals aged 4 and older with hyperphagia. CEO Anish Bhatnagar, M.D., says, “This milestone brings us closer to offering a new therapeutic for PWS. We extend our gratitude to everyone involved in the DCCR development program.” With Breakthrough, Fast Track, and Orphan Drug Designations, Soleno hopes for a Priority Review to expedite the FDA's process. Read the full press release at https://lnkd.in/gTXcnRpR. #SolenoTherapeutics #PraderWilliSyndrome #DCCR #FDA #MedicalBreakthrough

Congratulations to our own Dorothea Lantz (PWSA | USA Community Engagement Director) for winning Patients Rising's 2024 Fierce Advocate award! As many in our community know, this recognition is incredibly well-deserved. Dorothea is such an inspiration, constantly moving the needle in PWS and rare disease advocacy efforts. We are so proud of her for this achievement! Dorothea and other PWS advocates are back on Capitol Hill this week for the 2024 We the Patients Week in D.C. You can watch a livestream of the event at https://lnkd.in/gdXgk7yi.

Registration is now open for the 2025 International Prader-Willi Syndrome Conference in Phoenix Arizona! Prader-Willi Syndrome Association USA, FPWR and IPWSO are coming together to bring you the most up-to-date medical information, help and HOPE for parents and caregivers, a safe and fun space for your loved ones with PWS and their siblings, and a time to connect with other families. Space at the camps is limited and once it is full we will close registration for the camps. Mark your calendars and register early! * Register for the conference and camps at https://lnkd.in/g_3fBMpc