Burden of caregiving of individuals with GM1 and GM2 gangliosidoses in the United States: a qualitative study
This study showed a substantial humanistic burden with long-term impacts among the caregivers of individuals with GM1 and GM2 gangliosidoses. The findings provide important insights to enhance clinical care while advocating for the resources needed to improve caregivers' and patients' lives.
Authors
- Ruth Pulikottil-Jacob (ORCID: https://orcid.org/0000-0003-0630-960X)
- Christine Waggoner
- Chad Gwaltney (ORCID: https://orcid.org/0000-0001-8130-2024)
- Robert Krupnick
- M.B. Rodríguez (ORCID: https://orcid.org/0000-0002-5000-4080)
- Karli Heuer
- Diana Jussila
- Daisy Ng-Mak
- Nancy Gabriela Perez
Institutions
- CURE International UK (GB)
- Sanofi (France) (FR)
- AVEO Oncology (United States) (US)
- IQVIA (United States) (US)
- Sanofi (United States) (US)
- National Tay-Sachs and Allied Diseases Association (US)
- Sanofi (United Kingdom) (GB)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-11-20
- DOI
- https://doi.org/10.1186/s13023-025-04030-6
- Primary Topic
- Glycosylation and Glycoproteins Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Sanofi