Leading the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1 and Sandhoff diseases
Obtain a basic understanding of the research process. The NTSAD Scientific Advisory Committee closely monitors the scientific field and alerts…
Individual lysosomal storage disorders are classified as rare genetic disorders. However, when taken as a group, they are far more common.
Read through a friendly FAQ format to learn about leukodystrophies
Treating these diseases falls into two general categories: restoring the missing enzyme or decreasing the waste accumulation.
We often share literature which is written for the scientific community. Unless you are a researcher in the particular field,…