New call-to-action
New call-to-action
New call-to-action

FPWR Blog

Take Action for PWS Research: 4 FUN and EASY Ideas To Help You Get Started

There has never been a more critical time to keep research pushing forward. We need your help to raise the funds needed to advance PWS research. Whether you have never asked for donations or you are a veteran fundraiser, here are 4 fun and creative i...

PWS Registry Data: Thyroid Issues in PWS [INFOGRAPHIC]

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...

Topics: Research

SUMMIT SHEDS LIGHT ON IMPACT OF SLEEP DISRUPTION IN RARE DISEASES

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...

Social Skills and Cognition Challenges in PWS  [2020 CONFERENCE VIDEO]

In this 75-minute video experts Elizabeth Roof, Anastasia Dimitropoulos, Louise Gallagher, and Lauren Schwartz-Roth discuss what we have learned about social skills and social cognition challenges in PWS and what we can do to address the them so that...

Topics: Research

Neuren Pharmaceuticals Adds Prader-Willi Syndrome to Development Pipeline

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

Hoops to End Hunger - How One Family Turned March Madness into PWS Awareness

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

Sign-on to Our Letter to Make Sure YOUR Voice Is Heard By the FDA

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 Adds Synthetic CBD to Development Pipeline for PWS

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

FDA requires additional trial to support NDA submission for DCCR in PWS

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 pla...

Saniona Receives U.S. FDA Orphan Drug Designation for Tesomet in Prader-Willi Syndrome

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 ...