Leading the Fight to treat and cure
Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

9th Annual 'Fore' Jack Memorial Golf Outing

 

Piping Rock Club
Locust Valley, NY
Tuesday, September 12, 2017

Benefitting NTSAD's mission to lead the fight to treat and cure Tay-Sachs, Canavan and related genetic diseases and to support affected families and individuals in leading fuller lives.

For more information or to register by phone, please contact Sue Kahn at (617) 277-4463 or email at This email address is being protected from spambots. You need JavaScript enabled to view it..

 

Jack Kliger

Jack Alan Kliger was diagnosed with Canavan Disease when he was nine months old. Jack was given a life expectancy of three to five years. Jack passed away in April 2008 just shy of his 14th birthday.

Throughout his short life his family received support and encouragement by being part of the larger family of NTSAD and its members whose children fought similar diseases and similar battles. NTSAD offers parents information, explanations and resources as well as heartfelt support.

Your support will help NTSAD with their goal to eradicate Canavan, Tay-Sachs and similar diseases and also to help the children afflicted with these diseases and their families cope with the pain and suffering associated with these diseases.

What is Canavan?

Canavan is a progressive neurological genetic disorder caused by the absence of a vital enzyme known as aspartoacylase (ASPA). ASPA breaks N-acetylaspartate acid (NAA) into building blocks essential for building myelin. Myelin is a fatty membrane (also known as white matter) that forms a proactive coating around each nerve ensuring the nerve functions properly.  It is one of over 50 genetically inherited disorders known as Leukodystrophies Diseases.

What about research?

There are currently two gene therapy programs in development - Aspa Therapeutics licensed Dr. Guangping Gao's Canavan gene therapy work from the University of Massachusetts Medical School in Worcester, Massachusetts. They are currently working on a natural history study to have an even deeper understanding of the progression of Canavan; which will lead to establishing better endpoints in the upcoming clinical trial. The other program is at Rowan University under the purview of Paola Leone, PhD where she is exploring gene therapy using a different approach. More information is pending.

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