NTSAD and Tay-Sachs Gene Therapy Consortium Drives Key Initiatives in Gene Therapy


For over 50 years, NTSAD (National Tay-Sachs & Allied Disease) has been a world leader in the fight against Tay-Sachs (a progressive neurological disorder), Canavan and other related genetic diseases.  By funding global research and offering families with support and unity, NTSAD is truly making a difference in the lives of those suffering from genetic disease.

The Tay-Sachs Gene Therapy (TSGT) Consortium, a program managed within NTSAD, has been key in the initiatives driving gene therapy toward human clinical trials for effective genetic therapies.

In their recent July progress report, NTSAD provides an account of their recent successes in research and updates in gene therapy, including a recent article published in Forbes magazine which reported on a European’s impending approval of a gene therapy drug for the treatment of a rare genetic disorder.  It is currently awaiting final approval from the European Commission.

NTSAD is also in the planning stages of their second annual Day of Hope to be held on September 22, 2012.  Day of Hope is an opportunity for NTSAD member families and their communities to raise funding for research.  Over $10,000 was successfully raised in September 2011 from events held throughout the United States.

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