biostudies-literatureUnknown20(1)Rodriguez MB<h4>Background</h4>GM1 and GM2 (Tay-Sachs and Sandhoff diseases) gangliosidoses are rare, autosomal recessive, potentially life-threatening, disabling disorders characterized by progressive neurodegeneration, with no disease-modifying treatment. This qualitative study aimed to understand the humanistic burden of GM1 and GM2 gangliosidoses from caregivers' perspectives by expanding knowledge on the day-to-day responsibilities of primary caregivers and the impacts experienced while providing care and support.<h4>Methods</h4>Focus groups (90-minute duration) were conducted with caregivers (≥ 18 years) under three separate categories based on the age of the individuals with GM1/GM2 gangliosidoses either in-person (attending Annual National Tay-Sachs & Allied Diseases Association [NTSAD] ConferOrphanet journal of rare diseases597https://www.ebi.ac.uk/biostudies/studies/S-EPMC12632087biostudies-literatureBurden of caregiving of individuals with GM1 and GM2 gangliosidoses in the United States: a qualitative study.PMC12632087Rodriguez MBJussila DPerez NGNg-Mak DHeuer KKrupnick RPulikottil-Jacob RGwaltney CWaggoner CfalseBurden of caregiving of individuals with GM1 and GM2 gangliosidoses in the United States: a qualitative study.<h4>Background</h4>GM1 and GM2 (Tay-Sachs and Sandhoff diseases) gangliosidoses are rare, autosomal recessive, potentially life-threatening, disabling disorders characterized by progressive neurodegeneration, with no disease-modifying treatment. This qualitative study aimed to understand the humanistic burden of GM1 and GM2 gangliosidoses from caregivers' perspectives by expanding knowledge on the day-to-day responsibilities of primary caregivers and the impacts experienced while providing care and support.<h4>Methods</h4>Focus groups (90-minute duration) were conducted with caregivers (≥ 18 years) under three separate categories based on the age of the individuals with GM1/GM2 gangliosidoses either in-person (attending Annual National Tay-Sachs & Allied Diseases Association [NTSAD] Confer2025-01-01T00:00:00Z2025 Nov2026-06-05T17:27:55.996Z2026-06-05T03:07:03.367ZS-EPMC126320874126707810.1186/s13023-025-04030-6