News

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

The Foundation for Prader-Willi Research (FPWR) is presenting Harvesting Hope for PWS, a virtual “turkey trot” distance challenge to raise awareness about Prader-Willi syndrome (PWS), support research, and promote health and exercise. The nonprofit organization is encouraging supporters of all ages to log up to 100 miles walking, jogging,…

In a “significant subset” of  children with Prader-Willi syndrome (PWS), obstructive sleep apnea worsened after the start of growth hormone therapy, a study of patients in Australia found. “Our results offer support for the current advice to perform follow-up polysomnography [sleep breathing study] in children with Prader-Willi syndrome after…

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

Specific epigenetic modifications — chemical marks added to DNA that affect the way genes work — are found in people with Prader–Willi syndrome (PWS), and may affect how the disease manifests, a small study reported. These findings may help in understanding how genes contribute to the disease and open…

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

The Prader-Willi Syndrome Association (PWSA) USA has earned a Patients at the Heart award from Harmony Biosciences. Announced Wednesday on World Narcolepsy Day, which takes place every year on Sept. 22, the award was given to the project “Disordered Sleep: A Deep Dive for Parents and Practitioners.”…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

A new research project in Australia is investigating what underlies sleep-disordered breathing — an umbrella term for abnormal breathing patterns during sleep — often seen in children and adults with Prader-Willi syndrome (PWS). The goal, according to the researchers, is to better understand “breathing control stability” in children with…