PWS Awareness Month in May shines spotlight on the rare genetic disease

Supporters will celebrate and push efforts to speed PWS research, treatments

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Throughout May, supporters will celebrate Prader-Willi Syndrome (PWS) Awareness Month with a variety of educational, advocacy, and fundraising initiatives.

The goal of the month-long event is to raise awareness of the rare genetic disease and to support efforts that will accelerate PWS research and treatment development.

As it has in recent years, the Prader-Willi Syndrome Association USA (PWSA USA) offers 15 ways to get involved in the annual celebration.

Its suggestions for advocacy include reaching out to elected officials to promote legislative efforts, speaking at local institutions, and spreading the word using the association’s PWS Facts Toolkit and social media graphics.

The nonprofit also invites people to light their homes with PWSA USA’s colors — purple, pink, blue, and green — and send pictures to [email protected] so that they can be shared.

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Caregivers invited to share their stories about loved ones living with PWS

To make sure that individuals diagnosed with PWS take center stage, PWSA USA is also offering the Spotlight on Hope series, where caregivers are asked to submit a story that highlights a success achieved by a loved one living with PWS.

“Whether they took their first steps, had a great time at the school dance, made a shot in a basketball game, or are working towards a goal, we want to celebrate it all,” the association states on its awareness month webpage.

Also spotlighting PWS patients is the Rare Aware Art Share initiative, a virtual art show where PWS patients can share their work, offering them a platform to be seen and heard.

To mark the month, the association has also released the Voice of the Patient Report, which provides insights on the perspective of patients and families affected by PWS, and on aspects of the disease that may not be accurately captured in drug trials.

For the second year, PWSA USA is hosting a D.C. Fly-In, where 150 PWS advocates will head to Washington D.C. to learn about policies affecting the PWS community and to advocate for legislation that will benefit the community.

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Advocates will meet with elected officials for ‘Day on the Hill’ May 13-15

This year, the event will be held May 13-15, culminating in a “Day on the Hill,” where advocates will meet with elected officials to seek support for important legislative measures. Last year, a pair of legislators declared May 15 to be PWS Awareness Day in the U.S.

For its part, the Foundation for Prader-Willi Research (FPWR) is also suggesting a few ways to get involved through the month. Individuals can sign up to receive emails from the foundation with a daily PWS fact, which they are encouraged to share within their own community.

The foundation is also encouraging parents and caregivers of a person with PWS to update their survey responses in the Global PWS registry, the largest up-to-date natural history registry of the disease. It serves as an essential comparator group in clinical trials that are testing new therapies, so regular updates from affected families is necessary to keep it current.

The foundation indicates “nearly every dollar” they receive is donated by affected families and their networks. It offers templates, customizable graphics, and other resources for individuals wishing to start a fundraising campaign this month.

This year, a donor is matching all gifts up to $150,000. For every $1,000 a person raises, they’ll be entered into a lottery to win a pair of tickets to the annual FPWR conference, being held in Atlanta in September.

PWSA USA similarly provides ways to start a fundraiser, and offers new merchandise on its online store, with apparel and other items touting slogans such as “PWS is RARE but HOPE is NOT.”

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Supporters poised to observe PWS Awareness Month in May

‘Move It May’ efforts to light up landmarks orange in 30 UK towns

Elsewhere, PWSA UK and FPWR UK are joining forces to host “Move It May,” a fundraising effort to successively light up landmarks in orange in each of 30 U.K. towns.

The event asks individuals to record the time they spend being physically active with a goal of collectively traveling the entire 2,500 km path — or 59,000 Move It May minutes — by the end of May. Each town will light up orange as the milestone is reached.

On PWSA USA’s list of things to do this month is to sign a petition by May 23 urging the U.S. Food and Drug Administration (FDA) to act quickly on a future application seeking the approval of diazoxide choline (DCCR) extended-release tablets for PWS.

In Phase 3 clinical studies, Soleno Therapeutics’ investigational oral therapy demonstrated an ability to ease excessive hunger, or hyperphagia, a core symptom of PWS, and reduce fat mass and disease-related behaviors in PWS patients, 4 and older. Some of these results were recently published.

Earlier this week, the FDA granted DDCR its breakthrough therapy designation, making it the first PWS investigational therapy to earn such a status, which offers regulatory support and other incentives to expedite its development. DCCR also holds orphan drug and fast track designations in the U.S.

Among the incentives is the possibility that the FDA could grant priority review to the application seeking DCCR’s approval, an application Soleno expects to file by mid-year. Priority review would shorten the standard review time from about 10 months to 6 months. This is something the petition is seeking from the agency once the application has been submitted.