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 granted pediatric priority review status.
The extension is often a sign of the FDA’s commitment to fully evaluating all aspects of the New Drug Application (NDA) to ensure the treatment’s safety and effectiveness for our loved ones. This step underscores the complexity of rare disease drug development, where careful consideration is essential to delivering the best possible outcomes.
PWSA | USA is here for our community every step of the way and we are inspired by your resilience and hope as we navigate this journey together. Rest assured, the dedication of researchers, advocates, and families like yours continues to propel progress forward. If you have questions, concerns, or simply need someone to talk to, please don’t hesitate to reach out to us. Together, we will continue advocating for meaningful progress and supporting one another through this journey.
Let’s continue to hold onto hope and stay united in our mission to improve the lives of those living with Prader-Willi syndrome.
Sincerely,
PWSA | USA
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 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.
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.
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.
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.