Burden of caregiving of individuals with GM1 and GM2 gangliosidoses in the United States: a qualitative study.

ABSTRACT:

Background

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.

Methods

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] Confer

SUBMITTER: Rodriguez MB 

PROVIDER: S-EPMC12632087 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature