News

Soleno Therapeutics recently received fast track status from the U.S. Food and Drug Administration for diazoxide choline controlled-release (DCCR), its investigational treatment for Prader-Willi syndrome (PWS). DCCR is an extended-release, salt formulation of diazoxide, taken as a once-daily tablet. Diazoxide is a potassium channel activator, capable of crossing…

Inversago Pharma has raised $7 million to support the development of a new generation of cannabinoid-1 (CB1) receptor blockers for the treatment of Prader-Willi syndrome, type 1 diabetes, and other metabolic disorders. With the money, Inversago plans to advance its new compounds into clinical trials. CB1 inhibitors have…

Sleeping children with Prader-Willi syndrome (PWS) and severe obstruction of the upper airways — those between the nose and the larynx — go without sufficient breathing for longer periods when their apnea is worse, compared to children without the disease but with the same levels of obstruction, a study reports. The…

Zafgen biopharma and the nonprofit Foundation for Prader-Willi Research (FPWR) have launched “PATH for PWS,” a natural history study aimed at advancing the understanding of the medical history and medical events in patients with Prader-Willi syndrome (PWS). The non-interventional, observational study aims to document serious medical events experienced by Prader-Willi patients…

Treatment with anticonvulsant topiramate (sold under the brand names Topamax, Trokendi XR, and Qudexy XR) may help reduce food-related aggression and cravings in Prader-Willi syndrome (PWS) patients, a case report suggests. The research, “Topiramate in the treatment of Prader-Willi syndrome: A case report,” was published…

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…