National Tay-Sachs and Allied Diseases Association

Recent papers from National Tay-Sachs and Allied Diseases Association

1. Burden of caregiving of individuals with GM1 and GM2 gangliosidoses in the United States: a qualitative studyOrphanet Journal of Rare Diseases · 2025
2. Burden of caregiving of individuals with GM1 and GM2 gangliosidoses in the United States: a qualitative studyOrphanet Journal of Rare Diseases · 2025
3. GM1‐gangliosidosis: The caregivers' assessments of symptom impact and most important symptoms to treatAmerican Journal of Medical Genetics Part A · 2022
4. Family Driven Development of Treatments for Rare Pediatric Neurological DiseasesClinical Pharmacology & Therapeutics · 2021
5. A master protocol to investigate a novel therapy acetyl-l-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasiaTrials · 2021
6. A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 Gangliosidoses, and Ataxia Telangiectasia2020
7. Principles for interactions with biopharmaceutical companies: the development of guidelines for patient advocacy organizations in the field of rare diseasesOrphanet Journal of Rare Diseases · 2018
8. Natural History of Infantile GM2 GangliosidosisPEDIATRICS · 2011