On Tuesday, April 22, PWSA | USA and FPWR offered a PWS Community Town Hall for caregivers and guardians to hear more about people’s lived experiences with VYKAT XR. Experiences were shared from individuals who participated in the Phase 3 clinical trial of VYKAT XR, and attendees were able to ask questions of each other....
Hyperphagia, the relentless hunger that those living with Prader-Willi syndrome (PWS) experience, remains one of the most challenging and life-altering symptoms for individuals and families. But a promising investigational drug called ARD-101 is offering hope. In our April 29th episode of PWS United, Aardvark Therapeutics’ Dr. Tien Lee, M.D., CEO and Founder, and Dr. Manasi...
Date: Wednesday, April 16, 2025Time: 9:15 AM PT | 12:15 PM ETLocation: Virtual via ZOOM The Prader-Willi syndrome (PWS) community has reached a historic milestone – VYKAT XR (formerly known as DCCR in clinical trials) is now FDA-approved as the first-ever treatment for hyperphagia in PWS. To help families, caregivers, and healthcare providers understand what...
On June 28, 2024, Soleno Therapeutics announced the company officially submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (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, “Submission of the DCCR NDA to...
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...
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...
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...
This new website gives in-depth details about Acadia Pharmaceuticals COMPASS PWS Study, including a brief video about the study, eligibility for participation in the study, what to expect at study visits, and a research site locator. Click the button below to enter the website. ABOUT THE COMPASS PWS STUDY: The COMPASS PWS study aims to...
Harmony Biosciences has presented new secondary endpoint data concerning the use of pitolisant, an investigational drug, for the treatment of excessive daytime sleepiness (EDS) in Prader-Willi syndrome. According to a recent press release Harmony Biosciences released, the Phase 2 signal-detection study showed improvements in behavioral disturbances, especially in the higher-dose pitolisant group, as well as...
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...
Important information regarding this exciting milestone: Rare Disease Research (RDR), located in Atlanta, GA, is now welcoming children with PWS and their families to their clinicfor screening into this trial. The duration of active treatment in this study is 13 weeks. In a preclinical study in animals, physiological and behavioral symptoms were normalized within six...
You or someone you love could be part of developing new therapies for Prader-Willi syndrome. Learn about Gedeon Richter's research study KITE-PWS, also known as RGH-706-003, to evaluate an experimental drug for hyperphagia in people with Prader-Willi syndrome. The oral drug RGH-706 works by blocking melanin-concentrating hormone (MCH), which is a key part in the...
This morning, PWSA | USA received new information from Harmony Biosciences regarding its Phase 2 study of pitolisant. This study's purpose is to evaluate the safety and efficacy of the drug in patients with Prader-Willi syndrome (PWS). The company shared the following community-facing statement: "Today, Harmony Biosciences announced topline data on the primary outcome, excessive...
Contributed by PWSA | USA's Family Support Team The Centers for Disease Control and Prevention (CDC) has shown an increase in Respiratory Syncytial Virus (RSV) infections in multiple areas of the US https://www.cdc.gov/rsv/index.html. RSV infection can cause a variety of respiratory illnesses in infants and young children. It most commonly causes a cold-like illness but...
In an effort to help our PWS community better understand its SCOUT-015 trial, Radius Health has released several resources about the process of the study, trial sites, and about their drug RAD011 a synthetic cannabidiol oral solution, which is being studied as a possible treatment for hyperphagia and related behaviors in Prader-Willi syndrome. Click on...
PWSA | USA is happy to share the most recent news from Soleno Therapeutics regarding DCCR: This morning, Soleno shared that through continued dialogue with the FDA, the FDA acknowledged the data from a proposed randomized withdrawal phase of Study C602 would have the potential to address its concerns regarding the adequacy of the overall...
Contributed by Stacy Ward, MS Director of Family Support and Lynn Garrick, RN, BSN Medical/Research Coordinator Prader-Willi syndrome (PWS) is a rare neurodevelopmental genetic disorder that affects multiple systems in the body. There are many symptoms of PWS, including hyperphagia, behavioral challenges, hypotonia, incomplete sexual development, cognitive deficits, metabolic dysregulation, and several more. Hyperphagia is...
Radius Health has announced the locations for its RAD011 SCOUT-015 Clinical Trials. Below, you will find a downloadable map with each participating hospital and medical center around the country. The RAD011 SCOUT-015 studies are in various stages, depending on the location, from initiated to activated and ready to enroll. You can also find a comprehensive...
Read PWSA | USA Clinical Advisory Board's full Consensus Statement Here. Wednesday, October 20th is recognized as World Osteoporosis Day. Osteoporosis is a condition that commonly impacts individuals living with Prader-Willi syndrome, and is typically diagnosed in adolescence and adulthood. The cause(s) of the osteoporosis is not totally clear, but it is thought to be...
Written by: Merlin G. Butler, MD, PhD Historically, to confirm the diagnosis and molecular genetic classes in Prader Willi syndrome (PWS) required a stepwise approach using multiple methods needing more time and resources. Due to advances in genetic testing and availability of multiple analytical methodologies, a streamlined approach was developed and reported by Strom et...
(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,...
On behalf of Radius Health, Inc: Boston, Mass., July 22, 2021 — Radius Health, Inc. announced Wednesday, July 21, 2021 that it has recently received the written meeting minutes from a June Type C meeting held with the U.S. Food and Drug Administration (FDA) regarding RAD011, a synthetic cannabidiol oral solution. RAD011 is initially to...
FDA agrees to review additional data to determine adequacy for submission of NDA Soleno Therapeutics, Inc. provided an update on Tuesday, July 7, 2021 following a recent interaction with the FDA regarding the development of DCCR, a potential treatment for PWS. According to Soleno Therapeutics, on July 2, 2021 they received news from The FDA,...
Read Levo Therapeutic, Inc.’s Full Article HERE. CHICAGO, IL, July 6, 2021 (Newswire.com) – 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 the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for review and...
Jennifer Bolander has been serving as a Special Education Specialist for PWSA (USA) since October of 2015. She is a graduate of John Carroll University and lives in Ohio with her husband Brad and daughters Kate (17), and Sophia (13) who was born with PWS.
Over the years, as a special-needs parent navigating the Special Education system for Sophie, Jennifer became interested in learning as much as possible about that process. She began by attending PWSA (USA)’s first Wyatt Special Education Advocacy Training in March of 2013, and went on to complete a rigorous 9-month Special Education Advocacy Training course with the Council of Parent Advocates and Attorneys in May of 2015. This included successfully completing an advocacy internship with PWSA (USA). Jennifer is referred school cases by PWSA (USA)’s Family Support Counselors, and then works closely with parents and schools to review education records, assess the child’s situation at school, provide further information about how the syndrome affects the school experience, and, as needed, create improved IEPs and behavioral plans. Jennifer was excited to take on this role of helping parents in the PWS community to work collaboratively with school professionals and parents to create positive, effective learning environments for children with PWS across the country. Jennifer is a graduate of the WSEAT program.
Perry A. Zirkel has written more than 1,500 publications on various aspects of school law, with an emphasis on legal issues in special education. He writes a regular column for NAESP’s Principal magazine and NASP’s Communiqué newsletter, and he did so previously for Phi Delta Kappan and Teaching Exceptional Children.
Past president of the Education Law Association and co-chair of the Pennsylvania special education appeals panel from 1990 to 2007, he is the author of the CEC monograph The Legal Meaning of Specific Learning Disability; the more recently published books, A Digest of Supreme Court Decisions Affecting Education and Student Teaching and the Law; and the two-volume reference Section 504, the ADA and theSchools, now in its fourth edition. In 2012, he received Research into Practice Award from the American Educational Research Association (AERA) and the Excellence in Research Award from AERA’s Division A (Administration, Organization & Leadership). In 2013, he received the University Council for Educational Administration’s Edwin Bridges award for significant contributions to the preparation and development of school leaders. In 2016, he received the Education Law Association’s Steven S. Goldberg Award for Distinguished Scholarship in Education Law, and in 2017 he received the Council for Exceptional Children’s Special Education Research Award. Note:Perry’s website is perryzirkel.com
Evan has worked with the Prader-Willi Syndrome Association (USA) since 2007 primarily as a Crisis Intervention and Family Support Counselor. Evans works with parents and schools to foster strong collaborative relationships and appropriate educational environments for students with PWS.
Dr. Amy McTighe is the PWS Program Manager and Inpatient Teacher at the Center for Prader-Willi Syndrome at the Children’s Institute of Pittsburgh. She graduated from Duquesne University receiving her Bachelor’s and Master’s degree in Education with a focus on elementary education, special education, and language arts.
She obtained her PhD from Capella University in Education focusing her research on Prader-Willi Syndrome. Dr. McTighe is also a graduate of the William & Mary Law School’s Institute for Special Education Advocacy. Dr. McTighe is currently the chair of the Special Education Advisory Board for the Prader-Willi Syndrome Association-(USA). Amy is the Chair of PWSA (USA)’s Special Education Advisory Board.
Staci Zimmerman works for Prader-Willi Syndrome Association of Colorado as an Individualized Education Program (IEP) consultant. Staci collaborates with the PWS multi-disciplinary clinic at the Children’s Hospital in Denver supporting families and school districts around the United States with their child’s Individual Educational Plan.
Staci Zimmerman has lived in Denver, CO for the past 17 years. She lives with her husband and 5 year old daughter. Staci attended The University of Kansas, with a B.A in sociology and an M.ed in Special Education, with an emphasis in Autism Spectrum Disorder. Staci enjoys teaching fitness classes, and keeping a healthy, active lifestyle in Colorado.
Founded in 2001, SDLC is a non-profit legal services organization dedicated to protecting and advancing the legal rights of people with disabilities throughout the South. It partners with the Southern Poverty Law Center, Protection and Advocacy (P&A) programs, Legal Services Corporations (LSC) and disability organizations on major, systemic disability rights issues involving the Individuals with Disabilities Education Act (IDEA), Americans with Disabilities Act (ADA), and the federal Medicaid Act. Recently in November 2014, Jim retired.
Before founding SDLC, Jim was the Executive Director of Advocacy, Inc., the Texas Protection and Advocacy program from 1989-2001. During that period, he managed the growth of the program from 34 staff to a staff of 94 and from a centralized operation with one office to a regionalized operation with eleven offices spread across Texas.
From 1981-1989, Jim worked for the Advocacy Center for the Elderly and Disabled (Louisiana’s Protection and Advocacy program) as its Legal Director. From 1979 to 1981 Jim worked as a staff attorney for the Louisiana Center for the Public Interest.
Jim graduated from Tulane University School of Law in 1977, and has a BA in Accounting & Economics from the University of Puget Sound.
Jim is the author of publications and training materials on the IDEA, the ADA, and Section 504, including: Keeping Students with Disabilities in School: Legal Strategies and Effective Educational Practices for Preventing the Suspension of Students with Disabilities – A Resource Manual (2014); Stopping the Schoolhouse to Jailhouse Pipeline by Enforcing Federal Special Education Laws (2006), co–authored with Rhonda Brownstein from the Southern Poverty Law Center.
Jim has served as lead or co-counsel in several major IDEA actions in Texas, Louisiana, and Mississippi on behalf of thousands of students with disabilities, including Angel G. v. Texas Education Agency; Luke S. v. Louisiana Department of Education and Mattie T. v. Mississippi Department of Education. The past decade, he also filed and favorably settled five systemic administrative complaints in Louisiana, Mississippi, and Florida. These systemic complaints were filed under IDEA’s state complaint procedures.
Jim has also made over two hundred presentations at statewide, regional and national conferences on IDEA, Section 504, and ADA issues. The past four years he has also served as one of the faculty members at William and Mary Law School’s week long Institute on Special Education Advocacy.
