Foundation for Prader-Willi Blog | News

First-Ever PWS Genome Project Gets Crowdfunded

A new pilot project will map the genomes of 50 people with PWS and integrate that information with the Global PWS Registry data — with support from donors around the world.

This first-ever PWS Genome Project seeks to help researchers better understand differences in PWS symptoms and severity from person to person as well as issues of safety and efficacy of treatments in each person.

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Fundraising for PWS — Panel [2019 CONFERENCE VIDEO]

In this 45-minute video, Sarah Peden, Director of One Small Step and FPWR's Community Development Director highlights the success of the PWS community in raising funds for PWS research. Sarah and parents of children with PWS share how they've gotten involved in supporting PWS research, generating hope for themselves and others. Click below to watch the video, or scroll down to read a transcript of the session.

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FPWR Receives award from the Moriah Foundation to support the development of a Mental Health Guidebook for Prader-Willi Syndrome

The Foundation for Prader-Willi Research is excited to announce the receipt of a financial award from the Moriah Foundation which will be used to support the development of a Mental Health Guidebook for Prader-Willi Syndrome (PWS). Mental health and behavior challenges affect a large percentage of individuals with PWS and their families. These challenges are often under-appreciated and yet the impact on families and individuals with PWS can be profound. 

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Levo Therapeutics Receives Fast Track Designation From FDA for Intranasal Carbetocin for the Treatment of Prader-Willi Syndrome

Levo Therapeutics has received a Fast Track designation from the FDA for intranasal carbetocin for the treatment of PWS. A Fast Track designation will expedite the review of carbetocin so that, if shown to be effective, the product will get to market faster. This is very important for PWS as currently there are no treatments for many of the most challenging symptoms of PWS, such as hyperphagia.

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Millendo Announces Successful Recruitment of Phase 2b Study of Livoletide

Millendo announced today they have successfully enrolled 150 patients into their Phase 2b/3 clinical study of livoletide, completing enrollment for patients ages 8 to 85. The trial will continue to enroll patients ages 4 to 7 years old, and a minimum of 44 lbs. With the study now fully enrolled, topline results are expected in the first half of 2020 which may support a New Drug Application (NDA) filing for livoletide.

The second part of ZEPHYR is a Phase 3 study that will recruit additional patients at the same clinical sites and consists of a six-month double-blind, placebo-controlled core period in which patients will receive livoletide or placebo followed by a six-month extension period in which all patients receive livoletide. The primary endpoint for both the Phase 2b and Phase 3 portions of ZEPHYR is the change in food-related behaviors using the validated Hyperphagia Questionnaire for Clinical Trials (HQ-CT).

 

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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. 

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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.

Topics: News, Research

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.

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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.  

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Anyone Anywhere Can Raise Funds to Speed Research for Prader-Willi Syndrome

Community members from coast to coast are finding easy ways to make supporting PWS research FUN! A 1-mile kids race, a friendly kickball tournament, an uncle's bike ride across America, and an individual Ironman are just four examples of our community rising together to raise critical funds for PWS research while doing what they love!

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