Trinity Partners, a leading global life sciences consulting firm, announced that their client, Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals, Inc., has published results from one of the first-ever research studies measuring the considerable burden of patients with familial chylomicronemia syndrome (FCS) and the impact the disease has on their activities of daily living and employment. The results, from an interim analysis of the largest survey in patients with FCS, were published in Expert Review of Cardiovascular Therapy.
FCS, also known as lipoprotein lipase deficiency (LPLD) or type I hyperlipoproteinemia, is a rare genetic disease characterized by extremely high levels of triglycerides and increased risk of other health complications, including pancreatitis. IN-FOCUS, a comprehensive web-based research survey of patients with FCS, was designed to capture the associated burden of illness and impact on quality of life. Sixty patients from the United States diagnosed with FCS participated; patients described multiple symptoms spanning across physical, emotional and cognitive domains.
An analysis of the survey data found that patients with FCS saw five physicians on average with FCS-related symptoms before receiving a proper diagnosis. Nearly all respondents reported frequent, debilitating symptoms leading to reduced quality of life. Respondents also indicated that FCS significantly influenced their career choice and employment status, and resulted in substantial work loss.
Learn more about the study results: http://bit.ly/2oNlzbE
Read Endocrine Today’s article on the findings: http://bit.ly/2oaDxDE