News

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

Stress levels of parents of children with Prader-Willi syndrome (PWS) increase as the children age, and are especially high during their teenage years, according to a study. The study, “Parenting stress in families of children with Prader–Willi syndrome,” was published in the American Journal of Medical Genetics.

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…

Through the end of the year, the Prader-Willi Syndrome Association USA (PWSA USA) is offering 24-hour family support services to everyone in the Prader-Willi syndrome (PWS) community. The association’s family support team will provide emergency access to information and support for urgent medical and behavioral emergencies. Those in need…

Starting growth hormone therapy before the first birthday has significant benefits over beginning treatment at an older age in children with Prader-Willi syndrome (PWS), an analysis found. Children treated early grew taller, with a height similar to healthy children…

A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…

In older children with Prader-Willi syndrome (PWS), the probiotic supplement BPL1 reduced abdominal fat, social withdrawal and depression, while also easing insulin resistance, a study reported. These findings are consistent with results…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

Sleep apnea — a potentially serious disorder in which breathing repeatedly stops and starts during sleep — affects more than 80% of children with Prader-Willi syndrome (PWS), according to the results of a Chinese study. Central sleep apnea or CSA, a type of the sleep disorder that’s…

The U.S. Food and Drug Administration (FDA) has provided some recommendations to Saniona regarding the development of Tesomet to treat Prader-Willi syndrome (PWS). In particular, the agency is advising the company to conduct a Phase 3 trial to follow the planned Phase 2b clinical trial, as well…