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Participants in the summit discovered common issues related to sleep disruption across different disease states that had not previously been recognized.

Harmony Biosciences has published a white paper sharing outcomes from a Summit on Sleep Disruption the company hosted in December 2020. For the first time, this summit convened a diverse group of people living with rare diseases, caregivers and leaders of advocacy organizations who represented diseases in which sleep disruption has been recognized to discuss the role excessive daytime sleepiness (EDS) plays in their respective conditions. Theresa Strong, Director of Research Programs for FPWR, was among the Summit participants.


The goals of the summit were to describe the physical, emotional, social and cognitive challenges faced by patients with EDS; the challenges they face in having this symptom diagnosed; and its overall impact and burden on daily functioning.


"We want to help raise awareness of how excessive sleepiness is prevalent across the spectrum of rare disease,” said Cate McCanless, Senior Vice President, Corporate Affairs & Policy. “EDS is much more closely associated with rare sleep disorders, like narcolepsy, but it can pose significant physical, emotional, social, and cognitive challenges for so many others with rare disorders who may not even realize it.”


Participants in the summit discovered common issues related to sleep disruption across different disease states that had not previously been recognized. These issues include behavioral challenges, reduced social and cognitive abilities, increased comorbidities, increased anxiety and depression, and an overall reduced health-related quality of life. The summit also highlighted challenges in sleep measurement data collection in special needs populations and a difficulty with diagnosis and treatment in comparison with symptoms more commonly associated or accepted as a part of their respective rare disease.


The white paper can be accessed here. Harmony Biosciences will continue to work closely with advocacy and patient organizations to share these findings with the broader rare disease community.


Harmony is currently enrolling people with PWS ages 6 to 65 years old for a Phase 2 study which will evaluate the safety and efficacy of pitolisant in treating excessive daytime sleepiness (EDS), behavior, and cognitive function. Learn more about the study here.


Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.