PATH for PWS (Paving the way for Advances in Treatments & Health for PWS) is a natural history study to advance the understanding of medical events in people with PWS.
PATH for PWS is a natural history study co-sponsored by Zafgen and the Foundation for Prader-Willi Research (FPWR) to advance the understanding of the medical history and serious medical events in people with PWS. The data is intended to inform development and clinical trial design for potential new treatments. There is no therapy provided as part of this study.
In addition to the main PATH for PWS study, PATH participants who live in the U.S. will be asked to consider participating in a sub-study that involves providing a blood sample for analysis. Providing a blood sample is optional, and those who choose not to provide a blood sample for the sub-study remain eligible to participate in the main PATH for PWS study.
Enrollment in the 4-year study is underway through the Global Prader-Willi Syndrome Patient Registry, which is powered by the National Organization for Rare Disorders’ (NORD) IAMRARE™ Registry Program. We are looking for at least 500 people with PWS aged 5 years and older to participate.
What are the criteria to participate in the PATH for PWS study?
Why is the PATH for PWS study so important?
What are the PATH for PWS study details?
What is involved in the blood test sub-study, and do I have to consent to this sub-study to participate in PATH for PWS?
What types of serious medical events should be reported through the PATH for PWS study?
What is the time commitment involved in PATH for PWS study participation?
Will I be compensated for participation in PATH for PWS?
Will I be compensated for participation in the blood test (D-dimer) sub-study?
What are the other benefits of participation?
What is a natural history study?
Theresa V. Strong, Ph.D.
Dr. Strong is a co-founder and Director of Research Programs at FPWR, a nonprofit organization that supports research to advance the understanding and treatment of PWS. She received a Ph.D. in Medical Genetics from the University of Alabama at Birmingham (UAB) and performed postdoctoral studies at the University of Michigan. After her postdoc, she returned to the faculty at UAB, where her laboratory focused on developing gene therapy approaches for cancer, and was a Professor in the Department of Medicine prior to joining FPWR full time. She remains an Adjunct Professor of Genetics at UAB. Theresa has four children, including a son with PWS.
Children’s Hospital Colorado
Shawn McCandless, M.D.
Dr. McCandless is the Section Head for Genetics and Metabolism in the Department of Pediatrics, University of Colorado School of Medicine and Children’s Hospital Colorado. He received his medical degree from Temple University and specialized in Clinical Genetics and Genomics and Clinical Biochemical Genetics. He was previously a Professor of Pediatrics and Genetics, the Director of the Center for Human Genetics, and the Director of the Center for Inherited Disorders of Energy Metabolism at Case Western Reserve University and Rainbow Babies and Children’s Hospital. Dr. McCandless is experienced in caring for and performing clinical trials in children with a variety of genetic disorders, including PWS.
University of Florida
Jennifer Miller, M.D.
Dr. Miller is a leading PWS clinician and an Associate Professor in the Division of Pediatric Endocrinology at the University of Florida. She received her M.D. from the University of Florida, and completed an M.S. in Clinical Investigation from the University of Florida in 2005. She has worked extensively in the area of PWS clinical research, including endocrine and metabolic aspects of PWS, sleep and brain development. She follows hundreds of patients with PWS and provides ongoing advice to patients on the most effective and newest management strategies and treatments for PWS.