Get regular updates to your inbox.
Radius Health has acquired the development and commercialization rights to a synthetic oral cannabidiol therapy known as RAD011, with plans to start a pivotal Phase 2/3 clinical trial in Prader-Willi syndrome (PWS) this year. Having finalized its acquisition deal with…
A remote play-based intervention program improved pretend play skills, cognition, and behavior in children with Prader-Willi syndrome (PWS), a small study shows. The preliminary findings support the validation of this remote approach in larger studies of a patient population with a high unmet need for effective behavioral interventions, researchers said. The…
The annual EURORDIS Photo Award contest is inviting people worldwide to visually express what life is like with a rare disease, while raising awareness through their work of these disorders. Submissions for next year’s awards are open until Jan. 31. The competition is organized by France-based EURODIS, an alliance…
Growth hormone (GH) therapy initiated at an earlier age and sustained for a longer period results in greater improvements in growth and body composition in children with Prader-Willi syndrome (PWS), according to data from a real-world study. These findings further support the current practice of starting GH treatment as…
A three-week program based on Mediterranean diet and exercise, and performed three times in six years, led to significant weight loss and reduced cholesterol levels in adults with Prader-Willi syndrome (PWS), an Italian study reports. The study, “Changes of Body Weight and Body Composition in Obese…
To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…
The frequency of skin picking and anxiety in people with Prader-Willi syndrome (PWS) depends on their specific type of genetic defects, an eight-year study found. Treatment with growth hormone (GH) therapy increased the prevalence of anxiety to a different extent in specific genetic groups, the data showed. The study,…
The Prader-Willi Syndrome Association (PWSA) USA, a nonprofit organization that supports education, advocacy, and awareness for those with the complex genetic disorder, announced that it will hold its first benefit concert, “Hope for the Holidays,” on Sunday, Dec. 13, at 7:30 p.m. EST. The free, virtual event — designed…
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…
The maternal SMCHD1 protein ensures genomic imprinting and the effective silencing of particular genes by protecting them from “gene-activating” proteins in the fertilized egg cell (or oocyte), according to a study in mice. Since SMCHD1’s gene silencing effects are known to contribute to Prader-Willi syndrome (PWS), these findings may help…
Get regular updates to your inbox.