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Research Blog

DCCR Clinical Trial Should Continue, Says Data Safety Monitoring Board

Based on the review of data from more than 50% of patients enrolled, the Data Safety Monitoring Board has recommended continuing a  phase III clinical trial of DCCR for PWS. The trial, called DESTINY PWS, is a randomized, double-blind, placebo-controlled study of once daily oral administration of DCCR versus placebo by Soleno Therapeutics, Inc. 

Stages of PWS Drug Development Overview [VIDEO]

Drug development is a long and costly process that can take up to 15 years and cost up to one billion dollars! In this 7-minute video, Dr. Theresa Strong discusses the stages of PWS Drug Development. Don't have time for the full presentation? We've captured the key points in the notes below.

Phase 2 Study Results Published: Diazoxide Choline Controlled-Release

In 2015 FPWR co-funded a phase 2 study to investigate diazoxide choline controlled-release in patients with PWS. Results from the study were recently published in PLoS ONE and are summarized below.

Saniona reports positive Tesomet Phase 2a clinical results in adolescent patients with Prader-Willi syndrome

In a phase 2a study involving 18 patients with PWS (9 adults, and 9 adolescents) Tesomet reduced body weight and improved BMI and reduced hyperphagia to very low levels. Tesomet appears to be highly efficacious in adult and adolescent patients and was safe and well tolerated in the phase 2a study. Data from the 2a study will provide guidance for the pivotal phase 2b/3 studies now being planned. 

Evaluating Exercise Programs for People with PWS: New Study

The Foundation for Prader-Willi Research and the Prader-Willi Research Foundation Australia are jointly supporting a new research project, led by Dr. Nora Shields of La Trobe University, evaluating the effects of exercise training in individuals with PWS.

A Call To Action: Why PWS Clinical Trials Need to Be Full

Treatments for Prader-Willi syndrome are urgently needed. Today, we are closer than ever to the treatments for which we have been waiting, but we need all hands on deck to ensure our efforts can take us to the finish line. Here's why.

PWS Receives Specific Disease Code to Track Medical Care and Outcomes

The National Center for Health Statistics has designated a specific ICD-10 code for Prader-Willi syndrome (PWS). Previously PWS was included as one of number of genetic disorders ICD-10-CM code Q87.1, which contains a large group of genetic disorders associated with short stature. However, the genetic causes, medical complications, and treatment of these disorders are very different.

Debunking the Myths of Clinical Trials

Nearly 500 people with PWS are needed this year to fill PWS specific clinical trials. These trials are incredibly important to the PWS community and are necessary in order to bring treatments for some of the most challenging aspects of PWS. Here, we debunk a few of the most common clinical trial myths. 

 

Pitolisant approved for treatment of excessive daytime sleepiness in adult patients with narcolepsy

Harmony Biosciences has announced that the U.S. Food and Drug Administration has approved WAKIX® (pitolisant) for the treatment of excessive daytime sleepiness (EDS) in adult patients with narcolepsy.  

Managing Challenging PWS Behavior at Home and School: 2019 Conference Highlight

Need help with behavior management? Struggling with behavior at school?