Research 

Research plays a hugely important role in our work, whether we are fundraising to support research throughout the world, working with pharmaceutical companies interested in bringing drug trials to the UK, or making sure our families in our community have opportunities to take part in vital research.


This page has two primary purposes:


  1. To provide updates on the latest developments in research to improve the lives of those living with their PWS
  2. To promote opportunities for individuals with PWS and their families to take part in research, whether this involves drug trials or taking part in studies into different issues relating to PWS - for example, behaviour, learning and development, or the impact of PWS on family members


Both of these have the shared goal of better understanding PWS and, ultimately, improving the lives of those it affects.



Research updates

Soleno Therapeutics announces positive statistically significant top-line results from randomised withdrawal period of study C602 of DCCR for PWS

Posted 23rd March 2023

Read more here.

Soleno Therapeutics provides corporate update and reports fourth quarter and full-year 2022 financial results

Posted 23rd March 2023


Biopharmaceutical company Soleno Therapeutics has provided a corporate update, and reported financial results for the fourth quarter and full-year ended December 31, 2022. This includes an update on Study C602 of DCCR (Diazoxide Choline) extended-release tablets for the treatment of PWS.


Read more here.


Liraglutide for the treatment of obesity and PWS in children and adolescents
Posted 8th February 2023


The results of a study into whether liraglutide was effective for weight control in children with PWS have been published. You can read a summary here, which also includes a link to the more detailed findings.


Soleno Therapeutics announces peer-reviewed publication of results from previously completed Phase 3 trial of DCCR for treatment of PWS

Posted 27th January 2023


Biopharmaceutical company Soleno Therapeutics has announced that results from the its previously completed Phase 3 Destiny PWS (C601) trial of DCCR (Diazoxide Choline) extended-release tablets for the treatment of PWS have been published in The Journal of Clinical Endocrinology and Metabolism (JCEM) and show that while DCCR did not significantly improve hyperphagia, it did so in participants with severe hyperphagia. Read more here.


Soleno Therapeutics announces initiation of randomised withdrawal study of DCCR for the treatment of Prader-Willi syndrome

Posted 24th January 2023


Biopharmaceutical company Soleno Therapeutics has announced that it has initiated a randomised withdrawal period of Study C602, an ongoing open-label extension study of DCCR (Diazoxide Choline) extended-release tablets for the treatment of PWS.


Read more here.


Radius Health and RAD011

Posted 3rd October 2022


We have been working closely with Radius Health to bring a trial of their phase 2/3 clinical trial to evaluate RAD011 – a cannabidiol, to the UK. The trial was due to begin in the UK this Autumn but we are very sad that this has now been cancelled. Radius has made the following statement:


"Following a thoughtful evaluation of Radius Health’s current programs and product pipeline, Radius has decided to end its phase 2/3 clinical trial to evaluate RAD011 as a potential treatment for Prader-Willi syndrome. During October, we will begin winding down SCOUT-015.


"Radius was founded with the goal of addressing unmet medical needs and improving the lives of patients through the development and commercialization of new treatments, and we understand how difficult this decision is for enrolled patients, their families, and their caregivers. We are working closely with our research partners to help ensure that those enrolled in the trial are supported during this closeout."


Soleno provides regulatory update on DCCR

Posted 20th July 2022


Biopharmaceutical company Soleno Therapeutics has provided an update following recent communications with the U.S. Food and Drug Administration (FDA) regarding the development of once-daily DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS).


Read more here.


Soleno DCCR update

Posted 14th June 2022


Biopharmaceutical company Soleno Therapeutics has presented new positive clinical data from its ongoing late-stage DCCR development program for the treatment of Prader-Willi syndrome (PWS). The data shows that patients demonstrated

positive hormonal, cardiometabolic and body composition changes following one year of treatment with DCCR.


Read more here.


Update on the Stay Calm Project

Posted 10th May 2022


The findings of research by the University of Birmingham into emotional outbursts and meltdowns in children and young people have now been published. The study, known as the Stay Calm Project, was led by Justin Chung, under the supervision of Dr Kate Woodcock at the Centre for Applied Psychology, University of Birmingham.


You can read the full article here, but a summary of the research highlights can be found here.


Liraglutide and weight loss

Posted 1st April 2022


A promising weight loss drug is available across the counter at chemists, but not currently approved in PWS and has side-effects that need further consideration. Therefore, our advice is to wait until the results of the PWS-Liraglutide specific study are available. Read more here.


Soleno DCCR update

Posted 24th January 2022


Biopharmaceutical company Soleno Therapeutics has provided a promising update regarding the development of its DCCR (diazoxide choline controlled release) tablets for the treatment of Prader-Willi syndrome.


Soleno had a meeting with the US Food and Drug Administration (FDA) in December to discuss whether data submitted by Soleno to the FDA in October 2021 was sufficient to support a New Drug Application (NDA) submission for DCCR, as well as looking at ways to gather further controlled clinical data to support an NDA.


The FDA has agreed that a further study, if designed appropriately, can involve participants currently enrolled in the extension study C602 into the DESTINY PWS Phase 3 trial – a move that Soleno says will significantly reduce the time and cost involved in obtaining the necessary data.


Soleno now expects to submit a study proposal shortly and, if it is accepted, intends to progress the study.


Read more here.


Research papers of interest


Research opportunities

Behavioural and Emotional Outcomes in Neurodevelopmental Disorders study (BEOND) 

Posted 10th March 2023


Parents and caregivers of children and adults with Prader-Willi syndrome are being invited to take part in a new survey. The Behavioural and Emotional Outcomes in Neurodevelopmental Disorders study (BEOND) study is a large-scale survey being run by the Cerebra Network for Neurodevelopmental Disorders. Find out more here.


Research projects we have funded

Below are recent research projects for which we have provided funding:


  • January 2023 – University of East Anglia - The Sibs Project - £25,649. A qualitative study into the experiences and support needs of unaffected siblings and parents of individuals with Prader-Willi syndrome, which is currently under way. Further information on the project can be found here.
  • August 2021/December 2022 – Queen Mary University of London – Dr Elena Bockukova - Genotype to phenotype: understanding how our genome leads to variability in the Prader-Willi syndrome phenotype - £20,000 (£10,000 in each of the months listed above).
  • August 2019 – University of Cambridge – Dr A Holland - Investigating visual attention to different cues and the impact of food stimuli in infants and young children in Prader-Willi syndrome - £7,000.
  • February 2019 – Cardiff University – Dr Tim Wells - Generating a novel model of ghrelin-null Prader-Willi Syndrome - £3,300.



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