News

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

Adults with Prader-Willi syndrome (PWS) have difficulty recognizing facial expressions, which may contribute to their poor social skills, a study reveals. The researchers found that people with PWS also show atypical, autistic-like features, depending on the specific genetic cause of the disease. These findings expose…

Telomeres — a marker of cell division and lifespan — were found to be shorter in young adults with Prader-Willi syndrome (PWS) than in healthy controls, which could be a sign of premature aging, a study reports. The study, “Evidence for accelerated biological ageing in young adults…

Early diagnosis and treatment of Prader-Willi syndrome may delay obesity and minimize the risk for associated complications, a study suggests. The study, “Early Diagnosis in Prader–Willi Syndrome Reduces Obesity and Associated Co-Morbidities,” was published in the journal Genes, and was led by researchers at the University…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to LV-101 (intranasal carbetocin), a treatment candidate for people with Prader-Willi syndrome (PWS). This designation is granted to promising therapies intended to treat serious diseases with unmet medical needs. It aims to quicken treatment development by…

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

A Phase 2b/3 clinical trial of livoletide, a treatment candidate for excessive eating in people with Prader-Willi syndrome (PWS), has completed recruitment of participants who are 8 to 65 years old. However, enrollment of patients from 4 to 7 years old…

Children with Prader-Willi syndrome (PWS) have high levels of a protein called LIGHT/TNFSF14, which may contribute to obesity and low bone mineral density (BMD), a study suggests. The research, “High levels of LIGHT/TNFSF14 in Prader-Willi syndrome,” was presented at the European Society of Pediatric Endocrinology 2019 meeting in…

Daily consumption of a common probiotic significantly lowered the percentage of abdominal fat, improved insulin resistance, and reduced social withdrawal in patients with Prader-Willi syndrome, a study has found. The research, “Effects of Bifidobacterium animalis subsp. lactis on children with Prader-Willi syndrome: A randomized, double-blind, placebo-controlled, crossover trial,”…

Treatment with growth hormone for eight years led to a lower spine curvature in children with Prader-Willi syndrome (PWS) who developed scoliosis, a preliminary Dutch study reports. The research, “Effects of eight years growth hormone treatment on the onset and progression of scoliosis in children with Prader-Willi syndrome,”…