News

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

Butterflies, ballerinas, and abstract flowers are featured among the winning artwork of this year’s Rare Artist contest, hosted by the EveryLife Foundation for Rare Diseases to heighten awareness about rare disease communities and salute the creativity of its members. Awardees will be able to display their art and…

Due to the COVID-19 pandemic, the Prader-Willi Syndrome Association (PWSA) in the U.S. has announced its upcoming 36th National Convention,  “Hope’s on the Horizon,” will be moving to an all-new virtual format.  The PWSA will provide patients and families within the…

Intranasal use of the hormone oxytocin for three months had positive effects on social and eating behavior in boys with Prader-Willi Syndrome (PWS), according to a small clinical trial.  The results suggest intranasal oxytocin also may be an…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

Inversago Pharma‘s INV-101 has been granted rare pediatric disease (RPD) designation by the U.S. Food and Drug Administration for the treatment of Prader-Willi syndrome (PWS). This designation is given to potential medications targeting serious diseases that primarily affect less than 200,000 Americans ages 18 or younger. If the…

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…