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Alexion Joins Patient Organizations Worldwide in Support of International Rare Disease Day 2015Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) joins the European Organization for Rare Diseases (EURORDIS), the National Organization for Rare Disorders (NORD) and rare disease patient and healthcare advocates around the world in recognizing and supporting Rare Disease Day 2015. Rare Disease Day, celebrated annually on the last day of February, is dedicated to elevating public understanding of rare diseases and calling attention to the special challenges faced by patients and the community, including the need for improved diagnosis and treatment. The theme of this year's Rare Disease Day, "Living with a Rare Disease," focuses on the daily lives of the patients, families and caregivers who are impacted by rare diseases and pays tribute to the millions of family members and friends who are living "day-by-day, hand-in-hand" with rare disease patients. Alexion supports these key objectives of EURORDIS and NORD every day through its mission to develop and deliver life-transforming therapies for patients worldwide who live with severe and life-threatening rare diseases. "On this eighth annual Rare Disease Day, and every day, we pay tribute to patients and families living with rare diseases, as we recognize the challenges they continue to face, including receiving an accurate and timely diagnosis and accessing effective treatment options," said Leonard Bell, M.D., Chairman and Chief Executive Officer of Alexion. "At Alexion, our deep commitment to patients, families and caregivers drives us to work harder every day to overcome these obstacles through disease education efforts and by partnering with governments worldwide to ensure patients have access to life-transforming therapies." As a part of its support for Rare Disease Day, Alexion is engaging in a range of initiatives spanning across North America, Europe, Latin America, Asia and Australia to raise awareness of rare diseases, elevate rare diseases as an important public health issue and highlight the impact of rare diseases on patients and their loved ones' lives. These activities include educational efforts involving policymakers and medical professionals, partnerships with local organizations and participation in local Rare Disease Day-related events. North America
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In addition to these global initiatives to support the rare disease community, scientists at Alexion are working to understand the underlying causes of rare diseases and to discover and develop breakthrough medicines to treat them. Alexion's development programs include several highly innovative therapeutic candidates, which are being investigated in severe and rare disorders across the areas of hematology, nephrology, transplant, neurology, metabolic disorders and inflammatory disorders. To learn more about Alexion's Research & Development programs, visit www.alexion.com/pipeline. For more information about Rare Disease Day in the U.S., go to www.rarediseaseday.us, and for information about global Rare Disease Day activities, go to www.rarediseaseday.org. About Rare and Ultra-Rare Disorders In the United States, a disease is defined as rare if it affects fewer than 650 patients per million of population.1 The European Union definition of a rare disease is one that affects fewer than five patients per 10,000 of population.2 In contrast, a disease is generally considered to be ultra-rare if it affects fewer than 20 patients per million of population3 (one patient per 50,000) - and most ultra-rare diseases affect far fewer people than this. Despite the very small numbers of patients they affect, the impact of these rare and ultra-rare diseases on patients, their families, and society is profound, as many of these conditions are severe, chronic and progressive, with significant premature mortality. Patients with severe and life-threatening ultra-rare diseases often live without hope, have no effective treatment options and may face premature death. About Alexion Alexion is a biopharmaceutical company focused on serving patients with severe and rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. This press release and further information about Alexion can be found at: www.alexion.com. [ALXN-G] References: 1. U.S. Food and Drug Administration. Definition of Disease Prevalence for Therapies Qualifying Under Orphan Drug Act: http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm135130.htm 2. REGULATION (EC) No 141/2000 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 December 1999 on orphan medicinal products. http://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32000R0141&qid=1421232987002&from=EN 3. REGULATION (EU) No 536/2014 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC. http://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32014R0536&qid=1421232837997&from=EN
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