News

Insys Therapeutics’ Phase 2 trial to evaluate the safety and effectiveness of its cannabidiol (CBD) oral solution in pediatric patients with Prader-Willi syndrome (PWS) will soon begin recruiting participants. More information about the study and dates of enrollment will be announced on the clinical trial’s official webpage, found here (NCT02844933).

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

Saniona obtained regulatory approval to initiate the second part of a Phase 2a study evaluating the investigative therapy Tesomet (tesofensine/metoprolol) in adolescents with Prader-Willi syndrome (PWS) after positive results from the first part of the study with adult patients. Prader-Willi is a genetic disorder that begins in childhood. People…

Patients with Prader-Willi syndrome (PWS), diabetes, and fat accumulation in the pancreas may benefit from a combined treatment of two therapies that treat diabetes: tofogliflozin (which has not yet been approved in the U.S.) and dulaglutide, according to a Japanese case report. The study, “…

Low serotonin levels in the brain are responsible for a greater prevalence of affective psychosis in patients with a type of Prader-Willi syndrome (PWS) known as maternal uniparental disomy PWS (mUPD PWS), according to a study. Serotonin, a molecule found in the brain, is often referred to as a “happiness…

The Prader-Willi California Foundation is hosting its 18th Annual Walking for Prader-Willi Syndrome events in April, May, and June this year to raise awareness about Prader-Willi syndrome (PWS), a rare disease that is also the most common genetic cause of obesity. Events are planned in Los…

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…

Soleno Therapeutics recently released an update on the company’s achievements during 2017 and plans for its investigational therapeutic for Prader-Willi syndrome (PWS), a condition currently without an approved therapy. One of the highlights for the next steps in PWS is the FDA’s agreement with Soleno’s for a Phase 3 clinical…

Rhythm Pharmaceuticals has acquired the rights to develop and commercialize RM-853, an investigational therapy currently in preclinical research for Prader-Willi syndrome (PWS). RM-853, formerly known as T-3525770, is a potent oral inhibitor of ghrelin o-acyltransferase (GOAT), the key enzyme involved in the body’s production of the active form of ghrelin,…

If Theresa Strong didn’t keep the family fridge locked, her 23-year-old son Daniel might very well eat himself to death. It sounds shocking, but that’s part of the daily routine for parents of children and young adults with Prader-Willi syndrome (PWS), a complex genetic disorder that affects about 1…