Leading the worldwide fight to treat and cure
Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Donate to the Canavan Research Fund

The Canavan Research Fund of National Tay-Sachs & Allied Diseases Association, Inc. (NTSAD) is a fund created to support Canavan research. Research grants made from this fund will be directed to Canavan research specifically, and will help defray travel-related clinical trial expenses when and if needed.

To learn more about Guangping Gao, PhD and his work on a gene therapy for Canavan Disease at the University of Massachusetts Medical Center in Worcester, Massachusetts:

To make a donation:

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Donations by Mail or Phone:
(617) 277-4463
Checks payable to NTSAD
2001 Beacon Street, Suite 204
Boston, MA 02135

To set up annual recurring donations, please contact us at (617) 277-4463.


Wishing Dylan a Happy Birthday!

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.