Research Blog

Drug Development, the FDA and the Role of the PWS Community [WEBINAR]

Where are we with respect to new treatments for PWS? What are our next steps and how can the patient community help ensure that treatments for PWS are made available as soon as possible? In this webinar, our panelists discuss:

FPWR and PWSA|USA Request FDA Apply Regulatory Flexibility

FPWR and PWSA|USA have submitted a petition letter to the FDA requesting them to apply regulatory flexibility and to review a New Drug Application for DCCR (diazoxide choline extended-release).

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...

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...

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 people with PWS can be more successful. The session includes Q&A from participants in the 2020 FPWR Virtual Conference.

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. 

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 boy...

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 submission for DCCR in PWS.) This decision could cost us years, or worse, the loss of a potential treatment for our loved ones.

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 the PWS community since a previous clinical trial on the compound had to be cut short.

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 placebo in the primary and key secondary endpoints...