Dear PWS Families, We understand that hearing about the FDA’s extension of the review period for DCCR (diazoxide choline controlled release) may bring a mix of emotions, from hope to concern. Please know that this is a normal and expected part of the FDA’s thorough process, especially for a rare disease medication that has been...
On Tuesday, November 26, 2024, Soleno Therapeutics shared an important development regarding the New Drug Application (NDA) for DCCR (diazoxide choline) extended-release tablets—a potential treatment for individuals with Prader-Willi syndrome (PWS) aged four and older who experience hyperphagia. The U.S. Food and Drug Administration (FDA) has extended the review period for this NDA, pushing the...
On October 8, 2024, Soleno Therapeutics provided a regulatory update on DCCR (Diazoxide Choline Controlled-Release) tablets, which are currently undergoing priority review by the FDA as a potential treatment for hyperphagia in individuals with Prader-Willi syndrome (PWS). Although the FDA has moved forward with priority review, it has chosen not to hold an advisory committee meeting at this time. We have...
THE APPLICATION DEADLINE HAS PASSED. WE ARE NO LONGER ACCEPTING OPERATION HOLIDAY CHEER APPLICATIONS. ————————————————————————————————————— Thanks to the incredible generosity of an anonymous donor, PWSA | USA is thrilled to bring back Operation Holiday Cheer for 2024! This heartwarming initiative helps spread joy to families in need by easing the financial burden of the holiday...
As part of our ongoing efforts to best serve the Prader-Willi syndrome community, we are introducing a new name for our podcast. Moving forward, PWSA | USA’s podcast will be known as PWS United! This change is based on valuable feedback we’ve received from the community. Some members are sharing there is occasional confusion between...
Exciting news for the Prader-Willi syndrome (PWS) community! Soleno Therapeutics has announced that the FDA has accepted their new drug application (NDA) for DCCR, a drug designed to treat hyperphagia in individuals with PWS aged 4 and older. This acceptance is a major first step, and the FDA has granted Priority Review, recognizing the potential...
Contributed by Barb Dorn, RN, BSN As I began my research looking at specific health issues in the aging adult with PWS, I soon learned that there was not much information on this topic. I did find a few articles that documented clinical evidence for early signs of aging. As far as dementia, I found...
Compiled by Barb Dorn, RN, BSN People with PWS are growing old. Many of this may be the result of our increased knowledge in supporting and caring for the person with PWS. We have learned to replenish hormone deficiencies and manage their diet and food security. We have identified critical health issues and know that...
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...
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...
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...
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...
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...
On May 1, 2024, the first day of PWS Awareness Month, the Voice of the Patient Report was officially filed with the FDA! This timely filing marks a significant moment for the Prader-Willi syndrome community. The report gives crucial insights that amplify the perspective of individuals and families affected by PWS and sheds light on...
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...
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...
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 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...
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. ...
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,...
Neuren Pharmaceuticals is pleased to announce their second site participating in their Phase II, Open Label, PWS Study (Neu-2591-PWS-001) is now open for screening! Important information regarding this exciting milestone: Two sites are now open to enrollment! Rare Disease Research (RDR), located in Atlanta, GA, and Uncommon Cures, located in Chevy Chase, MD (8 miles outside...
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!...
Due to the generosity of an anonymous donor’s gift, PWSA | USA is once again able to bring holiday cheer to families in need this holiday season! We will identify a select number of families to receive gift cards to be used to help ease the financial stresses of the holidays. To be considered, please...
As children, we always remember our favorite gifts bestowed at holiday times: a shiny red bike, a beloved doll, a new baseball glove. These gifts and the loved ones who gave them to us are forever etched in our memories now and always. As the holiday season begins and the new year approaches, PWSA | USA is...
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.
