Topics: Clinical Trials Opportunities
Neuren Pharmaceuticals is now enrolling children with PWS in a phase 2 clinical trial to measure the safety and efficacy of NNZ-2591. NNZ-2591 is a novel synthetic analog of cyclic glycine-proline (cGP), a metabolite of IGF-1. NNZ-2951 regulates the ...
Join us September 27–28 for our annual gathering of movers, shakers, thinkers, and doers at this year’s FPWR Family Conference! It's a unique opportunity to connect, learn, and grow alongside like-minded individuals who are dedicated to making a diff...
Topics: News
In this 30‑minute video, Patrice Carroll, Elizabeth Roof, and Dr. Deepan Singh discuss strategies for addressing behavioral and mental health challenges in children aged 5 and up with Prader-Willi syndrome.
Topics: Research
The PWS-Clinical Investigation Collaborative (PWS-CLIC) is a network of PWS clinical experts, formally established in 2021. The work of the PWS-CLIC is supported by FPWR. This network has expanded over the past two years and now includes 25 clinical ...
Topics: Research, PWS People
In this 10‑minute video, Caroline Vrana-Diaz, FPWR Research Project Coordinator, explains how the PWS CLIC is helping to improve standards of care for people with Prader-Willi syndrome.
Topics: Clinical Issues, Research
Schaaf-Yang syndrome (SYS) is an ultra-rare disorder that was first identified in 2013. It is caused by mutations in the MAGEL2 gene, a gene that is also deleted or inactivated in Prader-Willi syndrome (PWS). Because SYS is a relatively newly describ...
As Rare Disease Day approaches on February 29, we want to spotlight the extraordinary qualities of our children with Prader-Willi syndrome, celebrating their unique passions and contributions to the world. Our objective is simple: to inspire hope. We...
Topics: Stories of Hope
In this 85‑minute video, Dr. Christian Schaaf, medical director and department chair at the Institute of Human Genetics at the University of Heidelberg and visiting professor at the Baylor College of Medicine, explains our understanding of Schaaf-Yan...
A paper has just been published that provides new insight into the use of feeding tubes in babies with PWS, thanks to the participation of ~350 families who completed the “Feeding Tube Survey” in the Global PWS Registry. Drs. Sani Roy, Ann Scheimann,...
Topics: Research