KITE-PWS is an ongoing Phase II trial of RGH-706 for people with PWS ages 17 and up. The study is sponsored by Gedeon Richter, a Hungarian-based company with 120 years of drug development experience. They have produced more than 200 drugs in over 40 ...
Drug development for new therapies begins with research and discovery. To treat PWS (Prader-Willi syndrome) and SYS (Schaaf-Yang syndrome), we need to understand the differences between individuals with these conditions and typical individuals, right...
Acadia Pharmaceuticals has announced the addition of a new Phase 3 development candidate to its rare disease portfolio, ACP-101 (intranasal carbetocin), for the treatment of hyperphagia in Prader-Willi syndrome (PWS).
A special blog contribution from Stephanie McDade. Last week Gibson rode a tricycle. She pedaled and steered on her own for a good five minutes on the sidewalk of our city block. This is real progress for a 8-year-old who doesn’t walk independently a...
Topics: Stories of Hope
You won’t want to miss these speakers at the 2023 FPWR Family Conference this October in Denver, CO.
Topics: Parents, Adults, Children 0-3, Advocacy
A special contribution by guest blogger Heather Osterman. Max came into the world with a bang...or more accurately, a limp body, not breathing, and blue. I went from being induced, since Max was quite cozy where he was, to a long labor, to an emergen...
Topics: Stories of Hope
FPWR’s grant program focuses on supporting innovative, high risk/high reward research in its early stages, with particular emphasis on supporting projects that will lead to new treatments for PWS.
Topics: News
FPWR is pleased to announce that we have recently awarded 13 project awards totaling $1.963M to support research aimed at advancing our understanding of Prader-Willi syndrome (PWS) and developing much-needed treatments. PWS is a complex genetic disor...
Topics: Research
Mice have long been the preferred mammal model in research labs due to their cost, size, and tractable genetics. Their behavioral simplicity, however, limits a researcher’s options for studying the component features that may serve as surrogate measu...
