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 humani...
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| Hlavní autoři: | , , , , , , , , |
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| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
BMC
2025-11-01
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| Edice: | Orphanet Journal of Rare Diseases |
| Témata: | |
| On-line přístup: | https://doi.org/10.1186/s13023-025-04030-6 |
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