FPWR Blog

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.

Topics: News

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.

Topics: News

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.

Topics: News

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.

Topics: News

Soleno Therapeutics Receives Fast Track Designation from FDA for DCCR for Treatment of Prader-Willi Syndrome

Soleno Therapeutics has received an FDA Fast Track Designation for diazoxide choline controlled-release (DCCR) for the treatment of PWS. Soleno is currently conducting a Phase III clinical trial of DCCR for the treatment of PWS. FPWR provided partial funding for the Phase 1 clinical trial.

PWS Clinical Trials Alert: July 2018

Several NEW PWS trial opportunities are now available. FPWR maintains the latest clinical trial information so that you can stay abreast of trial opportunities. Some trials require in-person visits while others can be completed remotely. Some trials are testing new drugs while others are intervention (non-drug) or natural history studies, like the PWS Weight Study described below.

Topics: Research

NEW Tool to Help Measure Anxiety and Distress in PWS

This guest blog was contributed by Sara Cotter, CEO, Levo Therapeutics.

When developing new medicines to treat PWS, companies need to demonstrate meaningful improvements in PWS symptoms. For growth hormone, this was relatively straightforward because changes in height are easily measured. For behavioral or psychiatric symptoms in PWS, this becomes a far more challenging task.

As a community, we are so fortunate to have the Hyperphagia Questionnaire for Clinical Trials, also known as the HQ-CT. This hyperphagia questionnaire was originally developed by Elisabeth Dykens and colleagues at Vanderbilt University, and further adapted for clinical trials by Zafgen and RTI Health Solutions. It measures observable behaviors related to hyperphagia, with questions like: “How upset did the person generally become when denied a desired food?” and “How often did the person try to sneak or steal food (that you are aware of)?”

Topics: Research

PWS Registry Data: 88% Have Received Speech Therapy [INFOGRAPHIC]

Speech requires a complex combination of cognitive processing to quickly find and combine the right words, paired with the physical process of producing sounds. Since PWS impacts a variety of pathways that contribute to speech and sound development, it is not surprising that 88% of individuals within the Global PWS Registry have at some point received speech therapy. The factors affecting speech and communication in PWS include issues with developmental delays, auditory and cognitive processing, oral and facial muscle strength, high-arched palate, and low saliva production. With regard to the sounds of speech in PWS, characteristics often include unusual prosody, or melody of speech; difficulty with articulating words; hypernasality; a slow speaking rate; and flat or abnormal pitch and intonation.

FPWR Conference Spotlight: PWS Behavior Experts

For every FPWR Family Conference, we introduce new panels, topics or activities, and this year I’m excited to announce our first-ever panel with three of the world’s most relevant PWS behavior experts.

Moderator Lauren Roth will lead panelists Elizabeth Roof, Kate Anne Woodcock and Anastasia Dimitropoulos in a discussion on what they’ve learned in recent trials on behavior and what is being done to help us all overcome behavior challenges in our loved ones with PWS.

We’re thrilled to bring such a high-caliber group of PWS behavior experts together for the first time, and we’re equally excited about the other sessions they’re involved with throughout the conference.

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.

Topics: News

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