News

Children with Prader-Willi syndrome (PWS) have abnormally high blood levels of certain inflammatory molecules, which is significantly associated with behavioral traits and symptoms of the condition, a recent study has found. The study, “A pro‑infammatory phenotype is associated with behavioural traits in children with Prader–Willi…

Growth hormone therapy improves height and body composition while lowering body mass index (BMI) in people with Prader-Willi syndrome (PWS), according to a review of clinical trials. The study, “Growth hormone treatment in Prader-Willi syndrome patients: systematic review…

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 U.S. Food and Drug Administration (FDA) gave positive feedback to Saniona regarding its work to advance Tesomet as a possible treatment to aid weight loss in people with Prader-Willi syndrome (PWS). Saniona now expects to file an investigational new drug (IND) application, asking to…

Soleno Therapeutics’ investigational oral therapy diazoxide choline controlled-release (DCCR) failed to significantly lower excessive appetite, but it did improve body composition and behavior in Prader-Willi syndrome (PWS) patients, top-line data from a Phase 3 trial show. Notably, excessive appetite (hyperphagia) — the hallmark symptom of PWS —…

Vitamin D levels are lower among adults with Prader-Willi syndrome (PWS) that in other obese individuals, mainly because PWS patients tend to consume less vitamin D through their diet, a study suggests. The study, “The Sun’s Vitamin in Adult Patients Affected by Prader–Willi Syndrome,” was…

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.

Tracking the eye movements of children with Prader-Willi syndrome (PWS) may be a non-invasive, low-cost, and reliable method to assess their chronic desire to overeat, known as hyperphagia, a study suggests. The study, “Eye tracking as…

Severe fluid and calorie restriction induced rapid weight loss that was effectively maintained with liraglutide, according to a case report of a young woman with Prader-Willi syndrome (PWS). Titled “Successful rapid weight reduction and the use of liraglutide for morbid obesity in adolescent Prader-Willi syndrome,” the…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…