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

Research Guide For Families

Details

test tubes and droppersNTSAD is dedicated to finding a cure for Tay-Sachs, Sandhoff, Canavan, GM1 and all the related diseases. Since we were founded over 63 years ago by a group of concerned parents, NTSAD has been at the forefront of advancing and supporting breakthrough research through the Research Initiative and collaborative programs. 

An Introduction to Research

To understand research, it is essential to have a basic scientific understanding of the diseases, the research process, and the terminology. The following links provide this introduction.

Overview of the research process: Journey to a Cure

All about lysosomal storage disorders (Tay Sachs, GM-1, Sandhoff, Farber, Pompe and more)

All about leukodystrophies diseases (Canavan etc) 

Technology approaches

Tips for reading scientific literature 

Resources to help evaluate the validity of a research study

Research by Disease

There are so many exciting efforts going into research it would be impossible to name them all here. The following pages serve as a summary of the recent research efforts. Most studies are in the Translational Research stage, but clinical trials are on the horizon.

Overall

Tay-Sachs Disease

Sandhoff Disease

GM1

Canavan Disease

Farber Disease

Get Involved in Research

There are many ways to contribute research. Whenever we learn about a new and appropriate study that is recruiting patients, we update the list below. Participating in research is a personal decision, and not one which every family feels is right for them. If you are considering participating in a research study, you are welcome to call NTSAD to discuss the benefits and risks. Read here about the different natural history studies currently enrolling.

Other Involvement

Fundraising - You do not have to participate in a study to be involved with research. Raising money for research is a vital way for research to continue moving forward. Browse the Get Involved section to learn about fundraising and donations.
 
Tissue Donation - If a loved one has passed away and you would like to help research progress to help others fight the disease, you may consider tissue donation. This is a private and personal decision. Learn more about tissue donation.
 

NTSAD and Research

How is my fundraising money used? How do you decide which studies are worth funding? What else do you do? Read NTSAD and Research for answers and more.

Stay Informed

The best way to stay informed about research is to sign up to receive our monthly emails with research updates! Sign up on the contact us page.

Previous Research Update issues and recent news may be found in the Library and for news from around the Internet view News Feeds.

 

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