- the latest PWS news, community updates & research
RARE DISEASES AND COVID-19
RDI and COVID-19
Rare Diseases International (RDI) have today published their statement on COVID-19 and the impact on the lives of people with rare disorders and the response that is needed from Governments.
IPWSO, as a member of RDI, recognises that people with rare disorders, including people with PWS, are at risk during this time of the COVID-19 pandemic, not just because of the virus itself but also because services to families and to people with PWS may be disrupted. Also people with PWS, if they themselves become ill with COVID-19, must have access to the necessary health care regardless of ability to pay - universal health coverage is crucial at this time.
If you know of anyone with PWS who has developed COVID-19 please complete our survey so that we can better understand how this type of infection affects people with PWS and can have a better understanding about the treatment and support received wherever you are living in the world.
LATIN AMERICAN NETWORK
Newsletter of the Latin American network of PWS specialists / Red de Especialistas Latinoamericanos SPW
Our conference in Cuba last November helped to cement the links between PWS experts throughout Latin America. Here is the recent newsletter that was sent out to the Network. If you would like to get involved do let us know.
IPWSO IS HIRING!
Could you join our small team?
We are looking for a talented and motivated Project and Operations Manager to coordinate the development and facilitation of a series of Project ECHO® telementoring initiatives that will connect learners with an interest in PWS from around the world using Zoom technology. See our advert for all the details about the role and how to apply!
ADDRESSING THE ETHICAL & THERAPEUTIC CHALLENGES OF A RARE DISABILITY
European Conference of Rare Diseases (ERCD)
IPWSO was represented at the above conference which was due to be held in Stockholm in May but was instead held virtually. Below is the link to a poster IPWSO submitted under Theme 2 of the conference 'Our values, our rights, our future: shifting paradigms towards inclusion'. Our Poster is number 055 and seeks to illustrate the specific challenges faced by people with PWS because of their hyperphagia and other behaviours.
Soleno announces interim results of the trial of Diazoxide Choline Controlled Release (DCCR) for hyperphagia
Soleno has been conducting the Destiny trial to investigate whether DCCR reduces the hyperphagia in people with PWS. The study findings did not meet statistical significance for the primary endpoint (reduction in scores on the hyperphagia questionnaire), but the study did find significant improvements in a pre-specified subgroup of people with PWS with severe hyperphagia. Significant positive changes were also seen in two of three key secondary endpoints. An interim analysis of the ongoing open extension study (C602) has shown further reductions in hyperphagia after six months of treatment with DCCR. Whilst the non-statistical differences in the primary endpoint are disappointing there are other encouraging findings and Soleno are continuing with the open part of the study and they will be discussing the findings of this trial with the regulators. IPWSO would like to thank Soleno for its commitment to seeking a treatment for hyperphagia in people with PWS and look forward to the final results from the 602 study.
Survey: The use of restrictive practices in the care of people with Prader-Willi syndrome
We are working with the University of Cambridge, UK, on a study collecting people’s views on the best way to support healthy lifestyles for people with Prader-Willi syndrome, whilst ensuring their rights are respected. Please follow the link to read more about this and to take part online.
If you would like to take part in a consultation when the early results of the survey are out please let us know.
POSITIVE DRUG TRIALS
Saniona announce positive findings for their combination drug 'tesomet'
Saniona has announced positive findings for their combination drug 'tesomet' in a Phase 2 trial in people with hypothalamic obesity. Weight and waist circumference reduced in those receiving the active drug versus the placebo control. The drug was well tolerated. A trial is underway with people with PWS and let's hope that similar positive findings will be reported in due course. This is encouraging news and IPWSO would like to thank Saniona for their commitment to the PWS community and their investment in this field.
COVID-19 & PWS GUIDANCE
New IPWSO guidance on supporting a person with PWS during COVID-19
Our Family Care Board has written guidance to give you some ideas about how to tackle the many issues that we are currently living with during the COVID-19 lock down.
NEW PWS RESEARCH PUBLISHED
Latest PWS research
Our Clinical and Scientific Advisory Board has published a collated list of all PWS research papers published from January to March 2020.
MILLENDO THERAPEUTICS FINDINGS
Millendo Therapeutics have announced the findings from their phase 2b trial of livoletide to treat hyperphagia in people with PWS.
Sadly there was no difference between those on treatment and those on placebo. IPWSO would like to thank Millendo for their work and for their commitment to people with PWS during the course of the trial. Livoletide is an unacylated ghrelin analogue and one possible approach to treatment, other studies are on-going that focus on different pathways. These trials are essential to ensure that potential treatments are thoroughly evaluated and the medications used are safe. People with PWS, their families and support staff have all contributed to this and to other trials. By all of us working together treatments will be found
Prader-Willi Syndrome (PWS) is one of an estimated 7,000 rare conditions defined in Europe and Canada as any condition affecting less than 5 in 10,000 people and in the USA as any condition affecting less than 6.40 per 10,000 people. Rare conditions described as 'neurological', because the brain is involved, are rarer still. PWS is one such neurological condition. The link above will take you to a report that specifically focuses on rare neurological conditions and PWS is included in the review.
In preparing the report the author approached IPWSO and Tony Holland, in his capacity as President of IPWSO, and Dr Maithé Tauber, a French endocrinologist and international expert on PWS and a member of the IPWSO Clinical and Scientific Advisory Board were interviewed. Maithé was able to describe how services have developed in France, an example of a Country with a well-developed services for people with PWS.
One of IPWSO's roles is to help support and inform the development of services for people with PWS and their families in countries where services are limited or non-existent. These reports can be helpful when you are trying to encourage you Government to develop or improve services. We encourage you to read this report and to ask yourself how the findings in the report may be used in your country.
IPWSO is grateful to the PWSA (USA) for permission to adapt, for an international audience, the statement on COVID-19 that PWSA (USA) had previously prepared. The Chair of the IPWSO Clinical and Scientific Advisory Committee has agreed this adapted statement. If important new information becomes available we will change or add to this statement (last updated 26 March).
IPWSO APPOINTS CEO
IPWSO appoints Marguerite Hughes as Chief Executive Officer
The International Prader-Willi Syndrome Organisation is proud to announce the appointment of Marguerite Hughes as our new Chief Executive Officer.
Marguerite is based in Ireland and has a doctorate in Social Science from University College Cork. Marguerite has held senior voluntary and paid positions in non-profit organisations in sectors including international development, patient support and advocacy, homelessness, and educational advocacy for 20 years. Marguerite's first child was born in 2004 and diagnosed with PWS shortly after birth. Prior to becoming IPWSO CEO, Marguerite served on the IPWSO board for 6 years, most recently as IPWSO Vice President from 2016-2019.
IPWSO President, Professor Tony Holland said, “The appointment of Marguerite Hughes as CEO of IPWSO marks an exciting new phase in IPWSO's work and the Board of Trustees look forward working with her in supporting people with Prader-Willi syndrome and their families around the world.”
APPLY FOR IPWSO 2020 CONFERENCE FUND
Application form for IPWSO 2020 Conference Fund sponsored by Soleno Therapeutics and Friends of IPWSO (USA), a non-profit organisation
In order to share knowledge about Prader-Willi Syndrome (PWS) around the world IPWSO is offering grants to individuals or organisations who wish to organise local, national or regional Prader-Willi syndrome workshops, meetings, or conferences. This scheme is sponsored by Soleno Therapeutics and Friends of IPWSO (USA), a non-profit organisation.
The closing date for the receipt of applications is 29 February 2020. The successful applicant/s will be chosen by the IPWSO board whose decision will be final. Both successful and unsuccessful applicants will be informed by 15 April 2019.