Canavan Research Fund

Supports research on Canavan disease and help defray travel-related clinical trial expenses when and if needed.

The Canavan Research Fund of National Tay-Sachs & Allied Diseases Association, Inc. (NTSAD) supports research to identify effective treatments for Canavan disease. Research grants made from this fund will be directed to Canavan research specifically, and when needed will also help defray travel-related clinical trial expenses for affected patients.

What is Canavan Disease?

Canavan disease 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. Canavan disease is one of more than 50 genetically inherited disorders known as leukodystrophies.

Clinical Trials

There are currently two gene therapy programs for Canavan disease –

Aspa Therapeutics licensed Dr. Guangping Gao’s Canavan gene therapy work from the University of Massachusetts Chan Medical School in Worcester, Massachusetts.

The other program – Myrtelle, Inc. licensed the research of Paola Leone, PhD, and explores gene therapy using a different approach.

Thank you from the bottom of our hearts for your support!

Ways to Give

Credit Card

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Mail or Phone

(617) 277-4463

Checks payable to NTSAD.

Mailing Address:
2001 Beacon Street
Suite 204
Boston, MA 02135.

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