CAMBRIDGE, Mass., March 26, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing drugs to treat people with serious and rare diseases, convened the first FCS Connection Summit on March 24th and 25th in Amsterdam, The Netherlands. The meeting provided a forum for patient advocacy organizations working on behalf of familial chylomicronemia syndrome, or FCS, to connect with each other to build a stronger and more collaborative global community.
“We are consistently humbled and inspired by the experiences that these individuals living with FCS share with us, and we are honored to be part of their journey,” said Molly Harper, vice president, and global franchise head of cardiometabolics at Akcea. “Since Akcea’s inception, we have prioritized partnering with patients and physicians. Through connecting with both the medical and patient communities, we are able to further enhance our approach to patient support and therapeutic development.”
FCS is a rare, potentially life-threatening disease with multiple severe, daily and chronic manifestations that affect one’s ability to work and engage in activities of everyday living. It is estimated that there are between 3,000 to 5,000 people living with FCS worldwide. Due to its rarity and the variability in terminology used by physicians to refer to FCS in the past, it is under-diagnosed and many patients, caregivers and FCS advocates have not had the opportunity to meet others living with this disease. This meeting brought together advocacy leaders from Europe, Canada and the United States, most of whom had never met each other previously.
“As with many individuals who are diagnosed with a rare disease, people living with FCS often express feelings of isolation. This is compounded by the burden of living with a serious and often misunderstood disease that affects every aspect of their lives including their relationships with others and their ability to work,” said Alan Gilstrap, executive director of advocacy and policy at Akcea. “By connecting FCS advocacy leaders with each other, we can enable and empower them to become more skilled and effective in their work, share best practices and to build a community of FCS champions to increase disease awareness, diagnosis and understanding.”
Among the actions taken during the Connection Summit was the decision to enact the first Friday in November as Global FCS Awareness Day. This year, Global FCS Awareness Day will be recognized on November 2. The global FCS community will use this day to raise awareness of the disease, working together to increase testing and diagnosis, and to share resources and information with both patients and healthcare providers. More details about this year’s FCS Awareness Day will follow.
“The opportunity to connect with each other was tremendously valuable, as we unite around a common mission to increase FCS disease awareness and diagnosis, and to let every FCS patient in the world know there is a community here to support them in their journey. Together, we have a great deal to accomplish, and the FCS Connection Summit was a wonderful springboard to propel us forward in our advocacy work,” said Jill Prawer, chair of the LPLD Alliance.
In addition to networking and best practice learning opportunities, Summit participants took part in skill building workshops, including Capacity Building for advocacy organization leaders and Persuasive Communications. People with FCS are unable to effectively metabolize fat and must adhere to a strict diet of no more than 10-20 grams of fat per day. As part of the Summit, Executive Chef Stéphanie Audet from Montreal’s Lov Restaurants demonstrated and prepared an FCS-friendly dinner for participants. Tips and recipes were provided for participants to replicate.
FCS is a severe, rare disorder characterized by extremely high levels of triglycerides, daily symptoms such as abdominal pain, and the risk of recurrent, potentially fatal, acute pancreatitis. People with FCS are unable to effectively metabolize large, triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides. There is no effective therapy available. Additional information on FCS is available at www.fcsfocus.com. For a full list of organizations supporting the FCS community worldwide, please click here.
Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious rare diseases. Akcea is advancing a mature pipeline of four novel drugs, including volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx. All four drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis' proprietary antisense technology. Volanesorsen is under regulatory review in the U.S., EU and Canada for the treatment of familial chylomicronemia syndrome, or FCS. Akcea is building the infrastructure to commercialize its drugs globally. Akcea is located in Cambridge, Massachusetts. Additional information about Akcea is available at www.akceatx.com.
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