Rare Daily Staff
Akcea Therapeutics announced that November 2, 2018 marks the inaugural Familial Chylomicronemia Syndrome (FCS) Awareness Day. The annual event will occur on the first Friday in November, as a global observance to build broader awareness of the challenges associated with the disease.
Familial chylomicronemia syndrome (FCS) is an ultra-rare, devastating hereditary disease that causes unpredictable and potentially fatal acute pancreatitis, chronic complications due to permanent organ damage, and a severe impact on daily living. It is estimated that there are between 3,000 to 5,000 people living with FCS worldwide.
“With the creation of an FCS Awareness Day, patients, caregivers and families have a new platform and opportunity to share their stories and to help others learn about FCS,” said Alan Gilstrap, executive director, advocacy and policy at Akcea. “We have had the privilege of hearing from so many patients about their experiences. Their stories highlight the continuing challenges that FCS presents every day and we are continuing our commitment to the lipid and FCS communities. Our hope is that more patients and caregivers can connect with each other, as we now know there are several benefits that connectivity can provide in FCS.”
In July, Akcea announced results of the CONNECT study providing many first-time assessments of the benefits of patient connectivity in FCS. Results showed a range of benefits when patients and caregivers connect in person or online, including improved perceptions of overall health, disease outlook, motivation to take care of health and emotional well-being. The study also found that connecting with other patients can positively impact the quality of life for people living with FCS.
“As part of the global FCS community, we are so pleased to recognize the first FCS Awareness Day, which is a great reflection of our mission to build awareness and provide support while also advocating for research that will lead to a treatment for FCS,” said Lindsey Sutton, co-president of The FCS Foundation. “As someone who knows personally how devastating FCS can be, I hope this day serves as a way to educate others about the symptoms and burden of the disease. Living with FCS can often feel isolating, and our goal is to help all patients realize that they are not alone in dealing with the challenges brought on by FCS.”
The FCS Foundation, along with members of the global FCS Community, invite patients, caregivers, clinicians, friends and family to participate on social media using hashtags #FCSAWARENESSDAY, #ACTIONFCS and #LIVINGWITHFCS.
FCS is an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase (LPL) and characterized by severe hypertriglyceridemia and a risk of unpredictable and potentially fatal acute pancreatitis. Because of limited LPL function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90 percent triglycerides. In addition to pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage, including chronic pancreatitis and pancreatogenic diabetes. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognition that affect their ability to work. People with FCS also report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog. There is no effective therapy for FCS currently available.
November 2, 2018
Photo: Lindsay Sutton, co-president of The FCS Foundation