News

Investigational Therapy May Alleviate Sleep Disorder, Cognitive Decline in Children with PWS

Pitolisant, an investigational anti-sleepiness therapy in the U.S., may help reduce daytime sleepiness and improve cognitive function in children with Prader-Willi syndrome (PWS), a case series suggests. The study, “Cognitive Improvements in Children with Prader-Willi Syndrome Following Pitolisant Treatment — Patient Reports,” was published in the Journal of…

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…

Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…

Exercise Improves Fitness in PWS Patients, Review Study Shows

Long-term exercise improves physical performance in patients with Prader-Willi syndrome (PWS), but immediate physiological response post-exercise appears unfavorable, a review of published studies shows. The study, “Physical exercise and Prader-Willi syndrome: A systematic review.” was published in the journal Clinical Endocrinology. PWS is associated with abnormal body composition and…

New Guidelines for PWS Treatment Implemented at Tennessee Clinic

A group of clinicians has established a series of guidelines for the treatment of people with Prader-Willi syndrome (PWS). The guidelines are already being implemented at Tennessee’s Prader-Willi Clinic at Monroe Carell Jr. Children’s Hospital at Vanderbilt. The new guidelines were described in the study, “A multidisciplinary approach…