News

A Phase 3 trial of Levo Therapeutics‘ treatment candidate LV-101 (intranasal carbetocin) is enrolling participants ages 7-18 with Prader-Willi syndrome. Patients are being recruited at the University of Florida and Vanderbilt University, with Levo anticipating additional study sites to open. Updates on trial sites can be found…

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…

The U.S. Food and Drug Administration has granted an orphan drug designation to Auris Medical for its intranasal betahistine product for the treatment of obesity associated with Prader-Willi syndrome. In addition to Prader-Willi syndrome, the company also has plans to expand the betahistine development program to include depression…

Aripiprazole, a medication used to treat the symptoms of schizophrenia and other psychotic disorders, is a potential therapeutic option for patients with Prader-Willi syndrome and psychosis, a new case report suggests. The study, “Clinical Usefulness of Aripiprazole Treatment in a Girl…

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…