Soleno Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has accepted their New Drug Application (NDA) and granted priority review for DCCR (Diazoxide Choline Controlled-Release), a treatment for patients with Prader-Willi...
70% of cases of Prader-Willi syndrome are associated with a “deletion” of 5,000–6,000 missing base pairs (bp) of DNA on the paternally inherited copy of chromosome 15. What are the biological consequences of the larger 6,000-bp deletion (typically ca...
Topics: Genetics and Imprinting, Research
A special contribution by guest blogger Lisa Matesevac. Welcome to the ATL! We are thrilled that our city will host the annual Foundation for Prader-Willi Research Symposium and Family Conference in September 2024! Together with my family and the Geo...
Topics: Parents
On Friday, August 2nd, the PWS Advocacy Coalition submitted a petition to the U.S. Food and Drug Administration (FDA), requesting the filing and priority review of the New Drug Application (NDA) for Soleno Therapeutics’ drug, DCRR. The petition signe...
The FDA has granted Rare Pediatric Disease Designation (RPDD) to ConSynance Therapeutics' new investigational drug, CSTI-500 for the treatment of Prader-Willi Syndrome in children and adolescents. This special status is granted to drugs and biologics...
Topics: News
You won’t want to miss these speakers at the 2024 FPWR Family Conference this September in Atlanta, GA. This year's conference has something for every parent traveling this PWS and SYS journey. Attend interactive workshops designed to provide practic...
Topics: Parents, Adults, Children 0-3, Children 3-6
Kathryn McGhee has been an active member of our PWS community for 22 years, participating in numerous letter-writing campaigns and PWS clinical trials and hosting over a dozen fundraisers for FPWR! As a proud mom to Hannah (who recently turned 23), K...
Topics: Stories of Hope, Parents
Just like the elite athletes competing in the Olympic Games, our community is passionate, dedicated, and persistent in their pursuit of victory! As our Olympians aim for gold, we are relentlessly working towards treatments for Prader-Willi and Schaaf...
Improving diversity, equity, inclusion, and accessibility (DEIA) in PWS clinical research is a priority for FPWR, and we are working to partner with experts and other patient groups working in this area. FPWR has recently joined the Rare Disease Dive...
Topics: News
