Topics: Research
When it comes to hormone imbalances in Prader-Willi syndrome (PWS), growth hormone deficiency and growth hormone therapy are at the forefront of the conversation. However, the levels of several other hormones are also more frequently different in PWS...
Harmony Biosciences has published a white paper sharing outcomes from a Summit on Sleep Disruption the company hosted in December 2020. For the first time, this summit convened a diverse group of people living with rare diseases, caregivers and leade...
In this 75-minute presentation, experts Elizabeth Roof, Anastasia Dimitropoulos, Louise Gallagher, and Lauren Schwartz-Roth discuss what we've learned about social skills and Prader-Willi syndrome. In particular, they discuss what we can do to addres...
Topics: Research
Neuren Pharmaceuticals has added Prader-Willi syndrome to their development pipeline for compound NNZ-2591 following a successful study in a pre-clinical model. The study showed compelling benefits for treating key symptoms of PWS.
Topics: Research
Nikol Maher, mom to Jack, turned her fear into determination using the family's love of sports. Jack was diagnosed with PWS when he was 3 weeks old. The news devastated our family and we could not begin to explain how fearful we were for our sweet bo...
Topics: Stories of Hope
In a disappointing decision, the FDA has stated that they will not review DCCR for the treatment of PWS without an additional controlled clinical trial. (Read more about this recent news in our blog: FDA requires additional trial to support NDA submi...
Radius Health has acquired a cannabidiol (CBD) compound and is preparing a PWS clinical trial using it. Radius Health believes that the drug has potential to reduce hyperphagia and improve anxiety symptoms in PWS. The news is of special interest to t...
Topics: Research
Soleno Therapeutics announced today that the FDA will require an additional controlled clinical trial to support an NDA submission for DCCR in PWS. This is despite pre-Covid data that showed statistically significant changes for DCCR compared to plac...
Saniona has received an orphan drug designation for their drug, Tesomet, for the treatment of PWS. Orphan drug designation is a special status granted by the FDA for drugs intended to treat rare diseases that affect fewer than 200,000 people in the U...