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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…

Loss of the paternal copy of Snord116, a gene associated with Prader-Willi syndrome (PWS), causes alterations in sleep and feeding responses in a mouse model of the disease, a study reported. Such changes result from an imbalance among different types of nerve cells in the animals’ hypothalamus, a…

Regular adherence to a careful dietary plan can help children with Prader-Willi syndrome (PWS) maintain a normal amount of visceral adipose tissue, the fatty tissue around abdominal organs, a study reported. The study, “Visceral adipose tissue resides within the reference range in children with Prader-Willi syndrome receiving nutritional intervention…

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, “…

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 —…