Rhythm Pharmaceuticals Inc., a company developing medicines for rare genetic disorders of obesity, has won FDA approval for its first product, Imcivree (setmelanotide), following a priority review. The drug is designed to restore a biological pathway that, when disrupted, can lead to constant hunger. The approval covers the treatment of three types of ultrarare...
The types of vaccines now available against COVID-19 use mRNA technology. This type of vaccine has been under development for many years and, unlike other immunizations, does not place a weak or inactive germ into the body, but instead teaches cells in our body to make an immune response that then produces antibodies which provide protection...
PLYMOUTH MEETING, PA and CHICAGO, IL, December 15, 2020 — Harmony Biosciences Holdings, Inc. (“Harmony”) (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the first patient has been enrolled in a Phase 2 trial evaluating the...
Respiratory illness in persons with Prader-Willi syndrome (PWS) can be extraordinarily complicated and the course of the illness is often more serious than it is for others. Pronounced hypotonia in PWS is a significant risk factor for respiratory illness with severe complications. In a recently released study by Dr.’s Miller and Thornton, parents of...
Food Security: Planning for the Holidays, Family Gatherings and Parties Holidays, family gatherings, parties and FOOD go hand in hand. With thoughtful planning and proactive preparation, these events can be successful, healthy experiences for everyone. It’s important that everyone is prepared and knows the game plan. Without a plan, the person with PWS may...
Finding a mental health professional who has experience working with individuals with Prader-Willi syndrome can be challenging. The following are some suggestions to help you with your search: Talk to your primary care provider. She or he might be able to recommend one or more clinicians who are familiar with PWS. Call your state...
What is newborn screening? Newborn screening is one of the most successful public health programs ever enacted, saving thousands of lives over the past 50 years. Newborn screening allows physicians to catch rare genetic conditions at birth and start treatment almost immediately following birth; it enables efficient diagnosis and access to treatments that can...
With childhood obesity presenting a significant threat to child health, the American Academy of Pediatrics (AAP) is recognizing National Childhood Obesity Awareness Month during the month of September. Early recognition and treatment of obesity has advantages. As we know in the PWS community, it is a daily struggle for most of our families. The...
A big help for me is having the refrigerator and pantry locked twenty-four hours a day, seven days a week. Having the pantry and refrigerator locked reduces my anxiety in or around those areas of the house. By implementing the lock-up tip, you will help keep your child healthy and minimize health issues. My...
Food restrictions in the home, although necessary for the safety and health of the family member with Prader-Willi Syndrome (PWS), can negatively impact siblings. The constant need to lock food adds intense stress and worry to siblings on several levels. For example, if the sibling forgets to lock access to food, this could result...
Prader-Willi Syndrome Association | USA (PWSA | USA) and the Foundation for Prader-Willi Research (FPWR) have partnered to conduct a study to measure interest in telehealth as a way of increasing access to care from Prader-Willi syndrome specialists. In addition, the project aims to better understand the demographic makeup of the Prader-Willi syndrome (PWS)...
There is little evidence in the literature regarding the incidence of gastroparesis in PWS and how it affects families in the PWS community. Within the Global PWS Registry, 13% of participants have been diagnosed with gastroparesis (N=739). Of those with gastroparesis, more than half were diagnosed under age 10. The severity of gastroparesis reported...
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.