Nation’s oldest patient advocacy organization rejoices as first human clinical trials for Tay-Sachs offer hope of conquering several rare diseases
November 27, 2012—BOSTON, MA—The National Tay-Sachs & Allied Diseases Association (NTSAD (http://www.ntsad.org)), the nation’s oldest patient advocacy organization, will honor President and founder of the EveryLife Foundation for Rare Diseases, Dr. Emil Kakkis (http://www.everylifefoundation.org/
“Emil knows what it means to bring hope to families suffering from rare genetic disease,” says NTSAD Executive Director Susan Kahn. “His vision that ‘no disease is too rare to deserve treatment’ reinforces NTSAD’s drive to identify treatments for children and adults affected by Tay-Sachs (http://www.ntsad.org/
NTSAD’s 55th anniversary also celebrates the first human clinical trials for a potential Tay-Sachs treatment, scheduled to begin in early 2013. The research (http://www.ntsad.org/
In addition to its research commitment, NTSAD supports more than 500 affected families and individuals worldwide while raising awareness of how to prevent Tay-Sachs and related neurodegenerative genetic diseases, which are caused by enzyme deficiencies.
“We are honored to recognize Emil Kakkis’ many accomplishments,”
Dr. Kakkis is also CEO and President at Ultragenyx. He is best known for his work over the last 20 years in developing novel treatments for rare disorders. At BioMarin, he guided the development and approval of three treatments for rare disorders.
Dr. Kakkis left industry in 2009 to start the EveryLife Foundation for Rare Diseases to accelerate biotech innovation for rare diseases. He initiated the CureTheProcess Campaign to improve the regulatory process for rare disease treatments through science-driven public policy. Ultimately, legislation was passed last July that will unlock life-saving treatments for rare diseases by increasing access to the FDA’s Accelerated Approval process.
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This 55th Anniversary Gala is the second of two 55th anniversary celebrations. NTSAD raised over $90,000 in Boston earlier this month with a tribute to former Genzyme CEO Henri Termeer, another pioneer in the biotechnology industry.
The National Tay-Sachs & Allied Diseases Association (NTSAD), the nation’s oldest patient advocacy organization, is dedicated to leading the fight to treat and cure Tay-Sachs, Canavan and related genetic diseases by funding promising research. The Tay-Sachs Gene Therapy (TSGT) Consortium, funded in part by NTSAD, carries out the most advanced gene therapy research and plans to begin the first human clinical trial in early 2013. NTSAD is equally committed to supporting affected families and individuals in leading fuller lives. Founded in 1957, NTSAD was a pioneer in the development of community education programs about Tay-Sachs disease and pre-marital carrier screenings. Today, more than two million people have been tested for Tay-Sachs disease, thousands of Tay-Sachs carriers have been identified and hundreds of healthy children have been born to high-risk couples. For more information, go to www.NTSAD.org.
Susan Kahn, Executive Director