Foundation for Prader-Willi Blog | Research

How Patients Can Partner to Speed Treatments for PWS [WEBINAR]

On May 15, PWSA-USA and FPWR jointly presented the webinar How Patients Can Partner to Speed Treatments for PWS. The webinar provided important information about PWS clinical trials including: what to expect when participating in a trial, what questions you should ask, and how to get the information you need to decide if a clinical trial is right for your loved one with PWS. The webinar also gave a brief overview of the trial opportunities that are available in 2018. You can watch the webinar in its entirety or read a summary of the webinar below.

Topics: Research

First Study Site Announced: DCCR for PWS Hyperphagia, Phase 3

Soleno has announced the opening of their first clinical trial site for a Phase 3 study of DCCR to measure the drug's efficacy for treating hyperphagia in PWS. That site is Seattle Children’s Hospital.

Topics: News, Research

Phase 2 Study on Oxytocin for PWS Now Recruiting

A Phase 2 Study of intranasal oxytocin (IN-OXT) is now recruiting patients for an 8-week double blind study to evaluate the safety and efficacy in pediatric patients with Prader-Willi syndrome. Data from the study will add to the current knowledge that intranasal oxytocin is an effective treatment for hyperphagia as well as other symptoms of PWS.

Topics: Research

PWS Registry Data: Anxiety In PWS [INFOGRAPHIC]

Anxiety is a common challenge for our loved ones with PWS. Recently, we reviewed responses in the Global PWS Registry related to anxiety. According to registry respondents, anxiety is an issue for 48% of individual with PWS ages 10 and up.

Topics: Research

Cannabidiol (BCD) Oral Solution for PWS: Phase 2 Clinical Trial Begins

INSYS Therapeutics recently initiated a Phase 2 clinical trial of its cannabidiol (CBD) oral solution for evaluation of safety and efficacy in pediatric patients with Prader-Willi syndrome.

The INSYS study will measure the effect of the company’s propriety CBD oral solution on behavior related to hyperphagia. The study is enrolling 66 patients ages 8 – 17 years old at 10 clinical sites in the United States, with results expected in the fourth quarter of 2019. The first trial site is now open. Click here for more details

Topics: Research

PWS Clinical Trials Alert | April 2018

The days we have been waiting for are now upon us! PWS clinical trials are coming quicker than you can imagine and we want to keep you up to date on these opportunities!  Sign up for our monthly Clinical Trials Alert to stay abreast of the latest trial opportunities and please share these opportunities with others that you think may be interested.

Topics: Research

Clinical Trials 101: What You Should Know About PWS Clinical Trials

It is an exciting time in PWS research, with multiple new clinical trials on the horizon this spring and through the end of 2018! There is already buzz throughout the community about upcoming studies, providing a wonderful opportunity for an open dialogue about the clinical trial process. FPWR does not advocate certain trials over others. We aim to provide resources for information, so that families are empowered with the knowledge and education to make the best decisions for themselves.

Topics: Research

Diazoxide Reduces Body Fat In Mice Lacking PWS-Region Gene

Many thanks to Dr. Wevrick for contributing to this blog.

A newly published study has shown that mice taking a diazoxide treatment lost weight, and that this occurred even in mice lacking the PWS-region gene, Magel2. The study, which is the first long-term study of diazoxide as a weight loss therapy in micewas published in the journal Molecular Genetics and Metabolism by Dr. Rachel Wevrick and her team at the University of Alberta in Edmonton, Canada. They have been measuring the ability of diazoxide to reduce body fat and increase endurance in mice. 

Topics: Research

PWS Places High Burden on Caregivers, New Publication Reports

PWS places a high burden on caregivers, impacting many aspects of their lives, according to a new publication in PLOS. The publication, High Levels of Caregiver Burden In Prader-Willi Syndrome, is the first stemming from the work of the PWS Clinical Trial Consortium (PWS-CTC). The study examined burden in 142 caregivers of children and adults with PWS living in the US using the Zarit Burden Interview (ZBI).

Topics: Research

Hip Dysplasia in PWS May Be More Common Than Thought, Study Suggests

Many people with PWS have at least one orthopedic (bone/muscle) problem. A recent paper examines the how often hip dysplasia occurs in children with PWS (spoiler alert — it may happen more often than was previously thought), and offers new recommendations for screening. The study was done at the Children’s Hospital Colorado, examining the records of ninety patients seen through their PWS Multidisciplinary Clinic.

 

Topics: Research

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