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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…

Children with Prader-Willi syndrome (PWS) as young as 4 are now eligible to participate in the ongoing Phase 3 trial testing diazoxide choline controlled-release (DCCR) tablets for PWS initiated by Soleno Therapeutics earlier this year. The original minimum age limit for enrollment was 8. Feedback from the U.S. Food…

Soleno Therapeutics announced the awarding of a patent for diazoxide choline controlled-release (DCCR) tablets, the company’s investigational therapy for Prader-Willi syndrome (PWS) being evaluated in a new Phase 3 trial. The patent covers “pharmaceutical formulations of diazoxide and diazoxide choline” to treat patients with poor lean body mass and a lean…

A variation in the growth hormone receptor (GHR) gene, specifically the lack of a coding sequence called exon 3, does not seem to increase the risk for scoliosis in Prader-Willi syndrome (PWS) patients, a small study reports. Exon 3 in the GHR gene was previously shown to be associated with increased growth…

A recent study from Australia revealed that patients with Prader-Willi syndrome (PWS) and Angelman syndrome can present autism symptoms, although the symptoms differ between the two conditions. According to the researchers, the findings have implications for treatment and intervention. Both Prader-Willi and Angelman syndrome are neurodevelopmental disorders that are…