FPWR Blog

In this 57- minute video, FPWR Executive Director Susan Hedstrom moderates as the FPWR research team provides an update on PWS research, achievements to date, and what we are doing to develop treatments for our loved ones with PWS. This session includes Q and A with conference attendees. Panelists include research team members Theresa Strong, Nathalie Kayadjanian, and Lauren Schwartz Roth and FPWR CEO John Walter. Click below to watch the video, or scroll down to read a transcript of the session.

Gillian Segall is an 18-year-old high school senior from Chicago, who loves to swim and take care of animals and also happens to have PWS. In this inspiring 15-minute interview, Gillian kicks off the 2019 FPWR Family Conference in New Orleans. Click below to watch the video, and scroll down for a full transcript.

In this 50-minute video, Theresa Strong and other FPWR research team members sum up the presentations given at the 2019 PWS Research Symposium. This overview provides a great synopsis of PWS research developments and work in progress, including PWS genetic therapy research, caring for people with PWS, managing clinical complications, newborn screening, drug development, pretend play, the PWS weight study, the Global PWS Registry, and clinical trials. Click below to watch the video. If you're short on time, scroll down for time stamps to find the portions you're most interested in.

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.

In this 51-minute video, Dr. Katy Chambers helps understand the IEP (Individualized Education Plan) process and succeed in the IEP journey. Katy Chambers is a school principal of nine years and mom to Daniel (age 7), who lives with PWS. She shares her journey maneuvering the educational system from both sides of the IEP table, providing strategies that have been helpful as a parent, tips that help school staff better understand our children’s needs, and action plans that help ease the day to day stresses of monitoring the safety of our kids in the school setting. She leads an interactive session, building on the collective knowledge and experiences of parents in the room. Click below to watch the video. If you're short on time, scroll down to the presentation outline to find the portions you're most interested in.

Have you ever wondered what is the optimal diet for a person with PWS? We may not be able to answer that question just yet, but in this 29-minute video, Dr. Anne Scheimann from Johns Hopkins shares preliminary data from her study on the Modified Atkins Diet (MAD) for PWS and on important changes it may have in the microbiome. They review diet strategies for PWS, go over the specifics of the MAD study, and share results related to weight changes and the microbiome. If you're short on time, scroll down for time stamps to find the portions you're most interested in.

Whether you are new to PWS or a veteran, its important to stay current on standards of care. In this 57-minute video, Dr. Jessica Duis describes standards of care for both children and adults with PWS, covering topics such as supplements, oxytocin, constipation and gastroparesis, hormone replacement and growth hormone therapy. Click below to watch the video. If you're short on time, scroll down to the presentation outline to find the portions you're most interested in.

Together, the Foundation for Prader-Willi Research, Angelman Syndrome Foundation and Dup15q Alliance are raising awareness of the similarities between Angelman, Dup15q, and Prader-Willi syndromes.

Gene therapies have the potential to provide transformative treatments in PWS but much is still unknown in regards to the feasibility of a successful treatment. In this 53-minute video, Drs. Stormy Chamberlain and Jim Resnick give a brief overview of PWS genetics, describe the current landscape of PWS genetic therapy, and discuss the feasibility of this therapy. This is followed by questions from the audience. Click below to watch the video. If you're short on time, scroll down for time stamps to find the portions you're most interested in.

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).