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...
Topics: Stories of Hope
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...
Topics: Stories of Hope
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...
Topics: Research
Brian Kalasek is both a father of a young son with PWS and VP of Special Risk for Mutual of Omaha insurance company. In this 76-minute presentation, he shares successful strategies for maximizing health insurance benefits and estate planning — inform...
Topics: Research
In this 88‑minute video, Dr. Deepan Singh discusses what anxiety looks like in PWS, what leads to it, and how can it be treated. Learn from this PWS mental health presentation, which concludes with Dr. Singh answering questions from the audience at t...
Topics: Research
In the last 5 years, more than 15 human clinical trials have taken place evaluating possible treatments for PWS. FPWR has been closely involved in each and every one: contributing the patient perspective to aspects of study design, funding or helping...
Topics: Research
