News

Millendo Therapeutics‘ treatment candidate livoletide failed to significantly reduce hunger and improve food-related behaviors in people with Prader-Willi syndrome (PWS) who are taking part in the pivotal ZEPHYR Phase 2b/3 clinical trial. Also, the treatment did not improve other measures, such as body fat, waist circumference,…

Parents and teachers attribute different severity to behavioral problems of children with Prader–Willi syndrome (PWS), according to a recent study. Although parents see food-related issues, such as repetitive speech and compulsive behavior as being more severe, teachers report that the impact of such problems on daily activities is higher. The study,…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

Altered gene expression underlies impaired function of brain areas regulating vision and motor control, which may be associated with behavioral issues in people with Prader-Willi syndrome (PWS) and other disorders, a study suggests. The study, “Central neurogenetic signatures of the visuomotor integration system,” was published…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

Obese people with Prader-Willi syndrome (PWS) should have access to exercise training programs different from those of obese people not affected by PWS, a case report suggests. The case study, “Differences in Aerobic Fitness between an Obese Adolescent with Prader-Willi Syndrome and Other Obese Adolescents and Exercise Training…

Small mutations in SNORD1116, a gene previously linked with typical manifestations of Prader-Willi syndrome (PWS), is associated with paranoia in women, a study shows. This finding reflects the greater incidence of mental health problems in those with PWS compared to the general population, scientists said. The study, “…

Children with Prader-Willi syndrome (PWS) may be particularly prone to metabolic alterations, including greater insulin resistance, which may be linked to increased risk for mental problems such as psychosis, a study suggests. The study, “Metabolic Parameters in…

Lipidio Pharmaceuticals has completed the first step of financing to support its development of GDD3898, a treatment candidate for Prader Willi syndrome (PWS) and other conditions associated with obesity. Investors in this series A funding include the Foundation for Prader Willi Research. “Prader-Willi syndrome, or PWS,…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…