- Created: Monday, 23 February 2015 20:02
The Plasma Protein Therapeutics Association joins more than 300 organizations in 56 countries in recognizing the 8th annual International Rare Disease Day on February 28.
Sponsored by the National Organization of Rare Disorders and Rare Diseases Europe, Rare Diseases Day seeks to raise awareness among the general public and policymakers about rare diseases, impacting lives all over the globe.
In the U.S., a disease is considered rare if it affects less than 200,000 people. In Europe, the definition is one that affects one in 2,000 people. Both organizations estimate that there are as many as 6,000-8,000 rare diseases, with about 80 percent being genetic, chronic, and often life-threatening. Those that are plasma protein disorders include bleeding disorders such as hemophilia, primary immunodeficiencies causing infections and cancer, alpha-1 antitrypsin deficiency causing lung and liver damage, and other rare diseases for which plasma protein therapies are the only available treatments.
The unique nature of rare diseases, including those treated with plasma protein therapies, requires constant vigilance to promote awareness and patient access to safe and effective therapies. PPTA and its members work tirelessly to ensure patients have timely and appropriate access to the best possible therapies. In both the U.S. and Europe, PPTA administers standards and certification programs, and is engaged in a broad range of regulatory issues, working to advocate and protect patient access to these therapies.
Today, these therapies enable patients with plasma protein disorders to lead active, productive lives. As recently as 20 years ago, patients with plasma protein-related conditions faced disability and a shortened life span. The progress has been remarkable, but there is much work to be done. Rare Disease Day 2015 shines a much-needed spotlight on the important needs of these patients throughout the world.
Media and Information
PPTA Proudly Supports World Hemophilia Day 2017 +
Register now for the 2017 Plasma Protein Forum! +
The 2017 Plasma Protein Forum will be held Tuesday, June 13 - Wednesday, June 14 at the Washington Marriott Georgetown in Washington, D.C.
Registration for the Forum includes access to all general sessions, the exhibit hall and all Forum breaks (you must be registered in order to participate in any of the Forum activities).Read More
The Hilfenhaus Award conferred to Prof. Farkas at the IPPC 2017 +
The 2017 Hilfenhaus Award was bestowed to Professor Henriette Farkas, MD, Ph.D., DSc, during IPPC 2017 for her work related to treating those suffering from Hereditary Angioedema (HAE) and working to improve treatments and patients’ quality of life. She is professor of allergology and clinical immunology at the Hungarian Angioedema Center at Semmelweis University in Budapest, Hungary.Read More