Harmony Biosciences recently met with three families impacted by Prader-Willi syndrome (PWS) to better understand their views on clinical trials for PWS symptoms including excessive daytime sleepiness.
A lot has changed since PWS was first identified and named in 1956, but one thing remains the same: The need to have additional treatments for people with this rare, genetic, multisystem, neurological disorder.
PWS has a range of associated symptoms that differ from person to person. For some, chief concerns may be hyperphagia, intellectual disabilities, or behavioral issues. For others, including the families featured in this article, the most debilitating symptom has been excessive daytime sleepiness (EDS).
These families say managing PWS symptoms may require a blend of therapies, interventions, and medicines tailored to the unique needs of each person living with PWS.
“There is not going to be one treatment – one medication or therapy – that is going to help every person with PWS,” says Kristi whose daughter Justice, age 20, has PWS. Kristi believes that the PWS community should have access to a range of treatments, including safe, effective, data-supported FDA-approved medicines. “Clinical trials are very important,” she adds. “It’s the only way that we’re going to find medications” to address PWS symptoms including those that may have previously been neglected, such as excessive daytime sleepiness.
Excessive Daytime Sleepiness: An Often-Overlooked Symptom of PWS
According to these families, excessive daytime sleepiness has had behavioral and social ramifications, disrupting personal growth and family dynamics.
“We’ve had to structure our lives around Victoria’s daytime sleepiness and be prepared to have a nap anywhere, at any time,” says Erin, mom of 7-year-old Victoria. “We’ve also had to say no to a lot of things.”
Another mother, Jennifer whose 19-year-old son Rocco has PWS with excessive daytime sleepiness, shared this: “Our family lives in two cars. We have a getaway car for whoever needs to take Rocco home, or we just don't go places or do things together.”
For instance, she says, “maybe I take Rocco [to his brother’s football game] for 15 minutes and then he's got to come home, because he can't sit on the bleachers that long and find a comfy spot because he needs to fall asleep.” Those are the hard moments,” she adds. “Rocco's not part of the family in those moments. We're a separated family in those moments. And those are the events where you think of a family sharing and building memories together.”
Click HERE for more about PWS and excessive daytime sleepiness at school.
Clinical Trials
By sharing their stories, these families hope they can inspire others to consider clinical trial participation. The potential availability of safe, effective, FDA-approved treatments for the various symptoms of PWS gives them hope for the future.
“I see our kids,” says Kristi. “I see our population. I see how amazing they are and how amazing they can be. But we need to find the right medications to help with the hardest parts of PWS.”
Watch a video from these families about their thoughts on clinical trials.
Learn more about a clinical trial studying an investigational medicine for excessive daytime sleepiness in people ages 6 years or older with PWS.
