Marisa Wexler, MS, senior science writer —

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…

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…

Measuring how electricity moves through the body could help detect low-grade inflammation in adults with Prader-Willi syndrome (PWS) without the need for a blood sample, a new study suggests. This non-invasive method could make it easier to detect increased inflammation — which has been linked to heart disease —…

Severe fluid and calorie restriction induced rapid weight loss that was effectively maintained with liraglutide, according to a case report of a young woman with Prader-Willi syndrome (PWS). Titled “Successful rapid weight reduction and the use of liraglutide for morbid obesity in adolescent Prader-Willi syndrome,” the…

The Foundation for Prader-Willi Research (FPWR) is funding nine projects that intend to improve understanding and help find treatments, including gene therapies, for Prader-Willi syndrome (PWS). In total, FPWR awarded a total of $912,251 in the first round of this year’s research awards, according to a press release.

Levels of the hormone irisin are elevated in obese people, but not in those with Prader-Willi syndrome (PWS), likely reflecting underlying differences in fat and muscle composition, a new study shows. The study, “Irisin levels in genetic and essential obesity: clues for a potential dual…

Parents of preschoolers with Prader-Willi Syndrome (PWS) tend to be relatively more stressed and overinvolved in interactions with their children, while the children themselves show less interest and social skill in these interactions, compared with their typically developing peers, according to a new study. The study, titled “…

A new funding deal will aid Saniona‘s development of Tesomet (tesofensine/metoprolol), a potential therapy to help control appetite and weight in people with Prader-Willi syndrome (PWS) and hypothalamic obesity, the company said. Saniona is planning to conduct a Phase 2b/3 trial, which the Denmark-based company…

Target enrollment of approximately 100 participants has been reached in a Phase 3 clinical trial evaluating the investigational oral therapy diazoxide choline controlled-release (DCCR) for reducing excessive appetite in people with Prader-Willi syndrome (PWS), Soleno Therapeutics has announced. Additional patients who are already scheduled to be screened…

Medix has submitted a new drug application to the Mexican food and drug administration — Comisión Federal para la Protección contra Riesgos Sanitarios, or COFEPRIS — seeking approval of tesofensine for the treatment of obesity. Tesofensine is one of two compounds that make up Tesomet, which Saniona…