Forest Ray PhD,  —

Children with Prader-Willi syndrome (PWS) experience significant reductions in their excessive hunger, anxiety and distress behaviors when treated with the lowest dose of Levo Therapeutics’ LV-101 (intranasal carbetocin) in a Phase 3 clinical trial, the company announced. While the highest dose under investigation (9.6 mg ) did…

The International Prader-Willi Syndrome Organisation (IPWSO) is seeking more responses to its COVID-19 survey evaluating the virus’ potential risks for people with Prader-Willi syndrome (PWS). The survey seeks to answer whether PWS patients might be at higher risk for infection, serious illness, and poor outcomes. Such…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

Growth hormone treatment given for years in childhood and then either restarted or continued as patients reach adult height shows sustained positive effects on body composition among young adults with Prader-Willi syndrome (PWS), including a decrease in fat mass, according to a Dutch study. The study, “…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.