Foundation for Prader-Willi Blog | News

PATH for PWS Study Highlighted by National Organization for Rare Disorders

In celebrating five years of success with its comprehensive patient registry, the National Organization for Rare Disorders (NORD) has called special attention to FPWR’s PATH for PWS study.

The study stems from improvements made to the NORD registry that allow greater collaboration and engagement among stakeholders and experts to accelerate the pace of research for rare conditions.

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Meet Lisa Matesevac, FPWR's PATH for PWS Study Coordinator

FPWR is happy to announce that Lisa Matesevac has joined our team as our coordinator for the PATH for PWS Study, facilitating families’ involvement in the study. PATH for PWS will help us understand the medical complications that people with PWS experience, and what factors can decrease or increase their risk. 

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Enrollment is Now Open for the PATH for PWS Study

The Foundation for Prader-Willi Research (FPWR) and Zafgen are pleased to announce that enrollment is now open for PATH for PWS, a natural history study intended to better understand serious medical events in Prader-Willi syndrome (PWS) and evaluate how PWS-related behaviors change over time. The data from this study is intended to inform the development and clinical trial design of potential new treatments for PWS. Those interested in participating can find more information about the study and how to enroll at www.PATHforPWS.com.

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PWS Clinical Trial Consortium and FDA Hold Key Meeting This November

The PWS Clinical Trial Consortium has recently been granted the unique chance to have a Critical Path Innovation Meeting with representatives of the FDA. The meeting will take place in November in Silver Spring, Maryland. This is a unique opportunity for the consortium and the PWS community to engage in discussions with the FDA on the progress made in the last three years by the consortium to overcome clinical trial challenges in PWS.

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FPWR Conference Spotlight: You!

We’re in the home stretch before the 2018 FPWR Family Conference, and the only thing missing is you!

From Oct. 4-6 in Las Vegas, we’ll bring together families, caregivers, researchers and PWS experts for education, networking and community-building. We’re also excited to welcome parents of individuals with Schaaf-Yang once again and USP7 syndromes for the first time.

It’s a powerful experience, to be sure, and it wouldn’t be the same without all of you! Here are some highlights of what to expect.

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FPWR Conference Spotlight: PWS Applied Behavior Analysis

We hope that every parent walks away from the FPWR Family Conference with new information they can use in their everyday lives, and our two sessions on PWS Applied Behavior Analysis (ABA) will be packed with practical tools.

Board Certified Behavior Analyst Cindy Szapacs and Associate Behavior Analyst Tony Chambers will lead separate sessions on ABA focused on dispelling myths and education advocacy, respectively.

For all parents who have faced or are facing behavior challenges, these PWS Applied Behavior Analysis sessions should shed light on a better path forward for you and your child.

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Finally! We have treatments for PWS in clinical trials. How have we gotten here?

We are in an exciting time in PWS research. More clinical trials evaluating new drugs for PWS are taking place this year than in the last 10 years combined! For the first time ever, families will be able to choose from multiple clinical trials enrolling patients across the United States. More than 500 patients will be needed to fill these clinical trials.

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FPWR Conference Spotlight: Schaaf-Yang Syndrome Workshops

For the second year in a row, we’re looking forward to holding Schaaf-Yang Syndrome Workshops at the FPWR Family Conference. Please join me in welcoming SYS families again this year!

In 2017, families with a diagnosis of Schaaf-Yang syndrome (SYS) gathered with us to share research information and insights. Schaaf-Yang syndrome is a genetic disorder that’s caused by a disruption of the MAGEL2 gene on chromosome 15. The same disrupted gene is also missing in PWS, which occurs when MAGEL2 and several additional genes on chromosome 15 are missing or inactivated.

Conference sessions marked “SYS Families” are either specific to SYS or will contain PWS information that’s also relevant.

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Theresa Strong Named to FDA Patient Engagement Collaborative

FPWR is thrilled to announce that Theresa Strong, our Director of Research Programs (and mom to Daniel, with PWS) has been selected to serve on the Patient Engagement Collaborative (PEC) by the U.S. Food and Drug Administration and the Clinical Trials Transformation InitiativeThe collaborative will share ideas on innovative ways for patients and the FDA to work together in the regulatory decision-making process.

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FPWR and Zafgen Partner for Natural History Study 'PATH for PWS'

FPWR and Zafgen have partnered to execute a four-year study that will advance the understanding of natural history and medical events in people with Prader-Willi syndrome.

The PATH for PWS study (Paving the way for Advances in Treatments & Health for PWS) is a non-interventional, observational study of serious medical events in PWS, intended to inform development and clinical trial design for potential new treatments for PWS.

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