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...
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. ...
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.
Perry A. Zirkel has written more than 1,500 publications on variousย aspects of school law, with an emphasis on legal issues in special education.ย He writes a regular column for NAESPโsย Principalย magazine and NASPโsย Communiquรฉย newsletter, and he did so previously forย Phi Delta Kappanย andย Teaching Exceptional Children.
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 Childrenโs Institute of Pittsburgh. She graduated fromย Duquesne University receiving her Bachelorโs and Masterโs degree in Education with a focus on elementary education, special education, and language arts.
Staci Zimmerman works for Prader-Willi Syndrome Association of Colorado as an Individualized Education Programย (IEP) consultant. Staci collaborates with the PWS multi-disciplinary clinic at the Childrenโs Hospital in Denverย supporting families and school districts around the United Statesย with their childโs Individual Educational Plan.
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.