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The safety and effectiveness of chenodeoxycholic acid treatment in patients with cerebrotendinous xanthomatosis: two retrospective cohort studies
OBJECTIVE: To evaluate the safety and effectiveness of chenodeoxycholic acid (CDCA) treatment in patients with cerebrotendinous xanthomatosis (CTX). METHODS: Two retrospective cohort studies were conducted in CTX patients who underwent CDCA treatment: one in the Netherlands (NL; CDCA-STUK-15-001) an...
Autores principales: | Verrips, Aad, Dotti, Maria Teresa, Mignarri, Andrea, Stelten, Bianca M. L., Verma, Sue, Federico, Antonio |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer International Publishing
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7160076/ https://www.ncbi.nlm.nih.gov/pubmed/31863326 http://dx.doi.org/10.1007/s10072-019-04169-8 |
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