PPTA Recognizes International Rare Disease Day 2012

ANNAPOLIS, MD – Plasma Protein Therapeutics Association (PPTA) joins over 300 organizations in 56 countries in recognizing International Rare Disease Day. This event, now in its fifth year, seeks to raise awareness among the general public and decision makers about rare diseases, impacting lives all over the globe.

There are over 1000 rare diseases including serious, chronic and often life-threatening plasma protein disorders. These include bleeding disorders such as hemophilia, von Willebrand disease, primary immunodeficiencies, chronic inflammatory demyelinating polyneuropathy syndrome, C1 esterase inhibitor deficiencies and alpha-1 antitrypsin deficiency, the majority of which are treated with therapies derived from human plasma. For these patients, there is no alternative treatment or chemical pharmaceutical available.

"People around the world coping with genetic diseases and disorders rely upon the life-saving therapies produced by our member companies. I am proud that PPTA has played a pivotal role in assuring patient access," said Jan M. Bult, President and CEO.

The unique nature of rare diseases, as well as plasma protein therapies, requires constant vigilance to promote awareness and protect therapeutic availability. PPTA and its members work tirelessly to ensure the quality and safety of these life-saving therapies. In both the U.S. and Europe, PPTA administers standards and certification programs, and is engaged in a broad range of regulatory issues, and works to advocate and protect patient access to these therapies. Bult said, "I am particularly proud of these programs which achieve safe and effective therapies." Additionally, PPTA collaborates with patient organizations through stakeholder meetings, publications and advocacy events. "Saving and improving lives of patients has been the hallmark of PPTA's advocacy focus as well as our quality agenda," Bult added.

Today, these therapies enable patients with plasma protein disorders to lead active, productive lives. As recently as twenty years ago, patients faced disability and a shortened life span. The progress has been remarkable, but there is much work to be done. Rare Disease Day 2012 shines a much-needed spotlight on the important needs of these patients throughout the world.

About the Plasma Protein Therapeutics Association
The Plasma Protein Therapeutics Association (PPTA) is a dynamic trade association that represents a unique sector of the biologics and biotechnology industry. PPTA member companies manufacture high-impact, high-value plasma protein therapies that make a difference in patients' lives every day throughout the world.

PPTA and its members focus on patient access to plasma protein therapies first and foremost. We work in tandem with more than 20 patient advocacy organizations to ensure that patients who infuse or inject these complex biologics have confidence in their safety and quality. Additionally, PPTA works globally with consumer groups, policymakers, and other key stakeholders on critical issues of patient access to care.

PPTA also administers standards programs, including the: International Quality Plasma Program (IQPP) and Quality Standards of Excellence, Assurance and Leadership (QSEAL): the National Donor Deferral Registry (NDDR), and the Patient Notification System (PNS). PPTA’s talented, multi-disciplinary team of experienced, dedicated professionals devotes itself daily to providing value to its membership and making contributions towards improving and saving lives.

About Rare Disease Day 2012
Rare Disease Day 2012 is an international, awareness-raising event coordinated by EURORDIS and the National Alliances of Patient Organizations. Hundreds of patient organizations from more than 40 countries worldwide are organizing awareness-raising activities converging around the slogan "Rare but Strong Together."

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