News

A year of treatment with DCCR (diazoxide choline controlled release) tablets, Soleno Therapeutics’ investigational therapy for people with Prader-Willi syndrome (PWS), safely led to sustained reductions in excessive hunger and disease-related behaviors, according to two presentations of data from a Phase 3 trial and its open-label extension. Soleno announced positive findings…

This year’s Prader-Willi syndrome (PWS) Awareness Month has launched, and will be celebrated with several educational and fundraising initiatives worldwide throughout May. Advocates will use the month-long event to raise awareness of the rare disease in the general community, and also among healthcare professionals, lawmakers, and researchers. The aim…

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…

Scoliosis — an abnormal sideways curvature of the spine — has a high prevalence among people with Prader-Willi syndrome (PWS), and becomes increasingly more common with older age, a study highlights. In fact, more than 80% of the PWS patients in the study, who were followed for more than…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

Starting treatment with growth hormone early in life — before age 2 — was associated with better cognitive and behavioral outcomes for children with Prader-Willi syndrome (PWS) in a recent study out of Spain. “We found greater cognitive and adaptive performance in patients who had received early treatment,” its…

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…

Saniona will pause the Phase 2b clinical trials testing Tesomet for people with Prader-Willi syndrome (PWS) and hypothalamic obesity (HO). The decision “is not related to the safety or efficacy of Tesomet and is entirely due to funding limitations,” the company stated in a press release. The…

Impaired social responsiveness, an indicator of autism spectrum disorder (ASD), is prevalent among adults with Prader-Willi syndrome (PWS), and is associated with worse social functioning, according to a recent study. “Our results suggest that evaluating difficulties in social skills is fundamental for the global assessment of functionality,” the researchers…

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…