News

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…

This year’s Prader-Willi California Foundation (PWCF) Annual State Conference will focus on educating families, caregivers, and professionals about the rare and complex genetic disorder, the foundation announced in a press release. Taking place Oct. 27 at the DoubleTree by Hilton in Sacramento, California, the conference will also emphasize…

Discontinuation of growth hormone (GH) therapy in adult Prader-Willi syndrome (PWS) patients causes accumulation of abdominal fat and increases levels of bad cholesterol, researchers in Japan report. The study, “Visceral adipose tissue increases shortly after the cessation of GH therapy in adults with Prader-Willi syndrome” was published…

Treatment candidate diazoxide chloride controlled release (DCCR) may cause fewer and milder side effects than approved medication Proglycem (diazoxide suspension) in treating low blood sugar in Prader-Willi syndrome patients, as well as providing consistent benefits, according to Soleno Therapeutics’ clinical studies. The findings showed that DCCR temporarily raises glucose (sugar)…

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…