Your experience matters in the quest to find treatments! ​

Your input is important! ​

Whether your child has (or had) the infantile or juvenile forms of Tay-Sachs, Sandhoff, GM1 or Canavan, or if you are affected by the Late Onset form, your participation is critical. We ask for you to share as much information as possible.​

Privacy is of the utmost importance; therefore, your information will remain private and de-identified. This data will be an immensely important tool for understanding our community and will greatly impact the path of clinical trial readiness. The FDA looks for as much of this data as possible as they consider approving clinical trials.​

Join the global community and share your experiences that are vital to the advancement of research toward effective and safe treatments.​

REGISTER with the PIN​

We hope you take some time to share your or your child's patient experience. It may seem overwhelming at first, but you should be able to move through it quickly and at your own pace.

You can fill the PIN out as much as you can, save it, go back to it when you have time and finish it, so it doesn't need to be all done at once.

Why do it? ​

Understanding how the disease impacts the child physically, and the whole family emotionally and socially, is important in making the case for a potential treatment to move forward to clinical trials and beyond. All experiences are welcome - past and present. And for our families outside of the U.S., you are welcome to register as well. Invitae has covered all the regulations in Europe so it is safe to participate in this PIN.​ You will also be notified of upcoming and open trials and studies.

There is only one way to get this information, and that is your willingness to share.  If you have any questions, please email Diana Pangonis with NTSAD here.​

How to register:​

​Step 1. Register your family or yourself on the respective PIN sites. ​

GM2 Tay-Sachs and Sandhoff here
Canavan here
GM1 here

Step 2. Fill out Survey 1 – Diagnosis information​

Step 3. Fill out Survey 2 – General Health​

Step 4. Fill out Survey 3 – Family History​

Step 5. Fill out Survey 4 – Medical Care Survey​