News

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…

Central adrenal insufficiency, a deficiency in cortisol levels due to problems in the pituitary or hypothalamus, is rare in people with Prader-Willi syndrome (PWS), affecting 1.2% of patients, a study shows. Researchers, therefore, do not recommend routinely prescribing medications for central adrenal insufficiency to PWS patients — as…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…