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…
The Gairdner Foundation recently announced the 2018 Canada Gairdner awardees for $100,000 prizes that recognize important research and biomedical discoveries. Among the five laureates are Davor Solter and Azim Surani for their discovery of parent-inherited genomic imprinting, a mechanism that regulates gene activity and can induce diseases such as Prader-Willi…
Caring for patients with Prader-Willi syndrome (PWS) has a significant effect on caregivers’ quality of life, and caregivers are in need of better support, a recent study found. Despite the positive effects that growth hormone therapy has had for patients with PWS, including improving their height, strength, and sense of well-being, those…
Silent aspiration, a consequence of a swallowing dysfunction, was detected in infants with Prader-Willi syndrome (PWS) by fluorescent live video, a study found. The research was published in the journal Medicine (Baltimore)Â in a study titled, “Silent aspiration in infants with Prader-Willi syndrome identified by videofluoroscopic swallow study.”…
Obesity and insulin resistance are significant risk factors for developing type 2 diabetes in patients with Prader-Willi syndrome (PWS), highlighting the importance of regular monitoring of glucose, or sugar, levels in these patients. Severe obesity in childhood, which may develop into type 2 diabetes, is a common feature in patients…
Only 40% of orchidopexies — a surgery to correct undescended testes — in patients with Prader-Willi syndrome (PWS) are successful, compared to a high rate of success in the general population, according to a new study. Cryptorchidism is the failure of one or both testes to descend from the…
Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes that cause at least half of the 7,000 rare diseases currently known — was the focus of a discussion by three top New York geneticists. The Feb. 28 conference,…
Diazoxide helps reduce weight, body fat, and improve blood glucose levels and endurance capacity, in mice models of Prader-Willi syndrome (PWS), a study shows. The findings suggest that diazoxide targets cellular mechanisms that contribute to PWS development. In addition, the study provides further evidence demonstrating diazoxide’s potential to treat…
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