News

People with Prader-Willi syndrome (PWS) may have an increased risk for coronary artery disease (CAD), a Portuguese case study suggests. CAD is the most common type of heart disease. It is characterized by the narrowing and hardening of arteries that supply blood to the heart due to the buildup…

Adult patients with Prader-Willi syndrome (PWS) show a lack of physical activity and deficient amounts of calcium, vitamin D, and fiber, a small study reports. These results support interventions to reduce sedentary lifestyles and the introduction of supplements to combat dietary deficiencies in Prader-Willi patients. The study, “…

Results from a Phase 2 clinical trial show that Levo Therapeutics’ lead therapy candidate, LV-101 (intranasal carbetocin), can effectively reduce symptoms of Prader-Willi syndrome (PWS) in children, the company announced. The investigational therapy was found to decrease the patients’ uncontrolled urge for food and excessive eating, with a…

Patients with Prader-Willi Syndrome (PWS) have lower levels of the angiopoietin-like 8 protein (ANGPTL8), which may be a marker for liver disease, than individuals of similar age and size who don’t have PWS, research shows. The results were reported in the study “Circulating angiopoietin-like 8 (ANGPTL8) is…

A new study shows that alterations in body composition, appetite-regulating factors, cardiovascular disease markers, and bone density are common in adults with Prader-Willi syndrome (PWS). The study, titled “Body composition, adipokines, bone mineral density and bone remodeling markers in relation to IGF-1 levels…

A new video explains how Prader-Willi syndrome (PWS) develops, some of its challenges, and ongoing research — all in just under three minutes. Titled “What is Prader-Willi Syndrome?” the video is narrated by Susan Hedstrom, executive director of the Foundation for Prader-Willi Research (FPWR) and the mother of a…

Saniona‘s investigative treatment for Prader-Willi syndrome, Tesomet tablets, were well-tolerated in preclinical toxicology studies, the company announced. The positive results support the safe dosing of Tesomet in long-term Phase 2 and Phase 3 clinical trials, the company said. Results also revealed that Tesomet, a fixed-dose combination of tesofensine…

As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…

Biotech company Saniona recently announced that the Phase 1 pharmacokinetic study of a new tablet therapy candidate for Prader-Willi syndrome, called Tesomet (tesofensine/metoprolol), produced promising results in healthy men. Prader-Willi syndrome (PWS) is an inherited disorder that creates feelings of constant hunger, which often leads to obesity and type 2 diabetes.

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…