News

Treatment with Soleno Therapeutics’ diazoxide choline controlled release (DCCR) tablets reduced excessive appetite and lowered body fat in Prader-Willi syndrome (PWS) patients in a Phase 2 trial, the company announced. The latest findings were presented during the Obesity Society Meeting 2018 in Nashville, Tennessee, in the poster, “…

Prader-Willi syndrome (PWS) patients have earlier-than-normal changes in body mass — associated with an increased risk of obesity, metabolic syndrome, and type 2 diabetes — which can be delayed with growth hormone (GH) treatment, a study shows. The study, “Early adiposity rebound in patients with Prader-Willi…

Two nonprofit organizations are backing an extensive pilot screening study in Australia for four rare genetic disorders, including Prader-Willi syndrome, which could lead to large-scale screening. Along with a $100,000 contribution from the Victorian Medical Research Acceleration Fund, the Angelman Syndrome Foundation (ASF), and the Foundation…

Analysis of facial movements in response to light and sound stimulation during ultrasound scans may help diagnose Prader‐Willi syndrome in babies before they are born, a case report suggests. The study, “Comparing a fetus diagnosed with Prader‐Willi‐syndrome with non‐affected fetuses…

Differing levels of tiny RNA molecules called micro RNAs (miRNAs) could explain the distinctive features of obesity between patients with Prader-Willi syndrome (PWS) and individuals whose obesity is not related to a genetic disorder, according to a study. The study, “Differences in circulating microRNA signature in…

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate, or…

Lean muscle mass is a strong predictor of bone mineral density, or bone health, research that used Prader-Willi syndrome (PWS) as a genetic model to determine which factors influence such density reports. In fact, lean mass was found to be a more important factor than overall weight, age or sex.

Soleno Therapeutics presented data supporting the clinical benefits of its treatment candidate diazoxide choline controlled-release (DCCR) in patients with Prader-Willi syndrome (PWS). Specifically, researchers showed that DCCR reduces hyperphagia — an abnormally increased appetite and a hallmark of PWS — by triggering potassium channels in certain nerve cells. Moreover, DCCR was as…

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…

The National Organization for Rare Disorders (NORD) is celebrating five years of its IAMRARE Registry Program’s success, as well as the creation of new collaborations and models of engagement, including one for Prader-Willi syndrome (PWS) research. NORD is the leading nonprofit organization that represents the 30 million…