NTSAD Research Library

Explore Scientific Literature and Other Resources on Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff Diseases.

Over the years, National Tay-Sachs & Allied Diseases (NTSAD) has curated a collection of scientific literature and other resources on Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff diseases.

We’ll continue to add to this library so you can stay up to date on developments in the field.

You can search the NTSAD research library for:

  • Scientific articles and posters
  • NTSAD newsletters
  • Advocacy information and toolkits
  • Care guides
  • Infographics
  • Position statements


Antisense Oligonucleotide Therapy for the Nervous System: From Bench to Bedside with Emphasis on Pediatric Neurology.
1 file(s) 1268 downloads
General November 5, 2022
Patients’ view on gene therapy development for lysosomal storage disorders: a qualitative study.
1 file(s) 1419 downloads
General October 21, 2022
CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges.
1 file(s) 1043 downloads
General November 12, 2020
Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord.
1 file(s) 1402 downloads
General October 6, 2020
Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock.
1 file(s) 2015 downloads
General September 18, 2020
Families and Researchers Collaborate in Advancing Therapy: The Role of NTSAD, Together with Parents, in Driving Toward Treatments.
1 file(s) 1339 downloads
General February 28, 2017

    Submit Your Research

    If you have an article or paper you’d like to submit to the research library, please email Valerie Greger (vgreger@ntsad.org), our Research Director.

    Need Help?

    If you or a family member is affected by a rare disease and you’d like help reviewing scientific literature, please contact our Family Support Team by emailing Diana Jussila (diana@ntsad.org), Director of Family Services, or Becky Benson (becky@ntsad.org), Family Services Manager.