National Tay-Sachs & Allied Diseases (NTSAD) to honor Dr. Emil Kakkis at 55th Anniversary

First human trial for Tay-Sachs, Canavan and other rare enzyme deficiency diseases plans to begin in early 2013 thanks to NTSAD, the nation's oldest patient advocacy organization, which leads efforts to find a cure or treatment.
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Nov. 27, 2012 - PRLog -- National Tay-Sachs & Allied Diseases to Honor Dr. Emil Kakkis at 55th Anniversary Celebration

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 (, the nation’s oldest patient advocacy organization, will honor President and founder of the EveryLife Foundation for Rare Diseases, Dr. Emil Kakkis (, at a gala celebrating NTSAD’s 55th year of leading the fight to find a cure. The event will be held December 5, 2012 at The Newark Club in New Jersey 6:30 p.m. to 10:00 p.m.

“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 (, Canavan ( and other fatal and debilitating lysosomal storage diseases (”

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 ( is the result of NTSAD’s long-time focus on funding promising studies.

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,” says NTSAD President Kevin Romer.  “We are also proud that NTSAD is recognized as a leading non-profit patient group with a demonstrated commitment to family support, research, and education. NTSAD gives help and hope to thousands of individuals and families from all backgrounds who have been or are affected by fatal genetic diseases.”  

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.

For event tickets and additional information go to:

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

Media contacts:

Susan Kahn, Executive Director



Edna Kaplan

KOGS Communication


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Tags:Tay-sachs, Gene Therapy, Clinical Trials, National Tay-Sachs, Rare Diseases
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Location:Salem - Massachusetts - United States
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