FPWR Blog

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...

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...

A special contribution by guest blogger Lindsey Larson Elliot earned the nickname “hero” before he was even born. I was 20 weeks along with our triplets, and I had noticed our only boy (Elliot), hadn’t moved in quite some time. Concerned, I called my...

Tonix Pharmaceuticals has licensed technology using oxytocin-based therapeutics from the French National Institute of Health and Medical Research (INSERM) to treat Prader-Willi Syndrome. The co-exclusive license allows Tonix to expand its development...

The last day of February is Rare Disease Day. This special guest blog by Rebecca was originally published on Rare Disease Day in 2016, and we're republishing to share with you this year. If you're inspired by her story, please consider making a donat...

Soleno Therapeutics and Vanderbilt University announce a new collaboration which aims to discover and develop novel KATP channel activators with the potential to treat rare diseases. Soleno’s lead product candidate, DCCR, is a potent activator of KAT...

In this 60‑minute video, Patrice Carroll, director of PWS services at Latham Centers in Massachusetts, describes what typical PWS behavior is, ways it can be improved, and how to remove barriers to allow children and adults with PWS to thrive and ach...