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Understanding patient and parent/caregiver perceptions on gene therapy in Gaucher disease: an international survey
BACKGROUND: Gaucher disease is a rare, autosomal recessive genetic disorder. It is caused by a lack of sufficient activity of the lysosomal enzyme known as glucocerebrosidase, which leads to an accumulation of glucocerebroside, a fatty substance, in the spleen, liver, bone marrow, and rarely, the lu...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2023
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9824927/ https://www.ncbi.nlm.nih.gov/pubmed/36611195 http://dx.doi.org/10.1186/s13023-022-02576-3 |