“For most people living with a rare disease, management of their condition is often a significant challenge and can impact many aspects of their daily lives including the ability to perform at work or school or take care of their families,” said
Established in 2008 by EURORDIS, Rare Disease Day takes place on the last day of February each year, with the goal to raise awareness of many different rare diseases and their impact on both patients and caregivers. Akcea has several activities planned globally around Rare Disease Day this year, engaging employees as well as patients, patient advocates, families and caregivers affected by hATTR amyloidosis and FCS.
“hATTR amyloidosis has placed a significant burden on my family and me both mentally and physically, especially as my symptoms have progressed, which include severe numbness in my hands and feet, GI distress, substantial weight loss, dizziness and exhaustion,” said Chuck, a patient living with hATTR amyloidosis with polyneuropathy. “Not only did this disease impact every aspect of my daily life, my wife Sharell took on new responsibilities of caring for me, which also came with their own adjustments and challenges. I am honored to be supporting Akcea’s efforts around Rare Disease Day and to continue to raise awareness of hATTR amyloidosis and provide support to other patients and their families.”
Akcea, along with members of the global hATTR amyloidosis and FCS communities, invite patients, caregivers, clinicians, friends and family to participate in Rare Disease Day on social media using hashtags #RareDiseaseDay, #ShowYourRare, #ShowYourStripes, #hATTR, #amyloidosis, #ActionFCS, #LivingWithFCS and #RareReality.
ABOUT HEREDITARY TRANSTHYRETIN (hATTR) AMYLOIDOSIS
hATTR amyloidosis is a severe, progressive, and life-threatening disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, the heart and intestinal tract. The progressive accumulation of TTR amyloid deposits in these organs often leads to intractable peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. hATTR amyloidosis causes significant morbidity and progressive decline in quality of life, severely impacting activities of daily living. The disease often progresses rapidly and can lead to premature death. The median survival is 4.7 years following diagnosis. Additional information on hATTR amyloidosis, including a full list of organizations supporting the hATTR amyloidosis community worldwide, is available at www.hattrchangethecourse.com or by visiting www.hATTRGuide.com.
FCS is an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase (LPL) and characterized by severe hypertriglyceridemia (>880mg/dL) 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% 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 cognitions 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. Additional information on FCS is available at www.fcsfocus.com, and through
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