News

The Foundation for Prader-Willi Research (FPWR) is launching the first Prader-Willi syndrome (PWS) Genome Project, it announced. This project aims to unveil how variations in patients’ DNA influence the range and severity of PWS symptoms, and impacts response to different treatments. Initially, it is looking to enroll about…

New brain-like organoids, or “mini-brains,” that are specific to key regions and derived from patients could serve as models for studies of the underlying neurological and molecular mechanisms of Prader-Willi syndrome (PWS), researchers reported. Notably, the team created arcuate organoids generated from cells of PWS patients, which captured the arcuate…

A new video series from Saniona is highlighting the impact, on patients and caregivers, of Prader-Willi syndrome (PWS) and an associated condition called hypothalamic obesity. Called “Saniona’s Community Voices,” the series features people living with rare diseases, including PWS, along with their caregivers, and healthcare professionals. The…

In interviews, most caregivers of people with Prader-Willi syndrome (PWS) reported an easing in patients’ behavioral symptoms after 13 or more weeks of treatment with diazoxide choline controlled release (DCCR) tablets in a Phase 3 trial or its ongoing extension study. Interviews were given to 48 caregivers of enrolled patients…

From joining a mixology course to participating in fundraiser bingo, supporters globally are set to mark Prader-Willi Syndrome Awareness Month, observed each May. Awareness and education are crucial to heightening the recognition, diagnosis, understanding, and management of Prader-Willi syndrome (PWS), a genetic disorder characterized by weak muscles, slow growth,…

Bladder problems leading to urinary incontinence and other manifestations are common among children, adolescents and young adults with Prader-Willi syndrome (PWS), a study shows. The study, “High prevalence of lower urinary tract dysfunction in patients with Prader–Willi syndrome,” was published in the journal Neurourology and Urodynamics.

A planned Phase 2b trial of Tesomet in treating Prader-Willi syndrome (PWS) is being pushed back to later this year, following a request from the U.S. Food and Drug Administration (FDA) for more information on the medication’s manufacturing. “While we are disappointed to delay the initiation of our Phase 2b…

The excessive eating experienced by people with Prader-Willi syndrome (PWS) intimately affects the burden felt by their caregivers, according to a recent study. While this comes as no surprise to caregivers, the finding provides some of the first direct evidence of this relationship, which can inform future studies. The…

The Foundation for Prader-Willi Research (FPWR) and Saniona are working together to inform patients and their families of an upcoming Phase 2b trial evaluating Tesomet as a potential treatment of Prader-Willi syndrome (PWS), and to best ensure community involvement. Initiation of this study, which follows positive findings in a…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…