News

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

The Prader-Willi Syndrome Association and the Foundation for Prader-Willi Research (FPWR) have partnered to launch a survey assessing the use of telemedicine as a way of improving access to care from Prader-Willi syndrome (PWS) specialists. The online survey also seeks to evaluate the stress of caregivers within…

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…

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

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…

Structural changes in a brain region called the cerebellum are associated with behavioral problems in people with Prader-Willi syndrome (PWS), according to a study in Japan. The findings provided, for the first time, strong evidence supporting the cerebellum as a key contributor to impaired brain connectivity and behavioral issues…

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 —…

Children with Prader-Willi syndrome (PWS) whose disease is caused by a deletion in part of chromosome 15 may be more prone to obesity than others with non-deletion disease causes, a study suggests. Different gene expression in the brain likely explains the variability in patients from each group, researchers…

High protein meals induce the secretion of the insulin-stimulating hormone glucagon-like peptide 1 (GLP-1) in children with Prader-Willi syndrome (PWS), suggesting that these kinds of meals may help suppress appetite, a pilot study has found.  The study, “…

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…