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Severe homocysteinemia in two givosiran-treated porphyria patients: is free heme deficiency the culprit?
Givosiran is a novel approach to treat patients with acute intermittent porphyrias (AIP) by silencing of ∂-ALA-synthase 1, the first enzyme of heme biosynthesis in the liver. We included two patients in the Envision study who responded clinically well to this treatment. However, in both patients, th...
| Autores principales: | , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Springer Berlin Heidelberg
2021
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8195940/ https://www.ncbi.nlm.nih.gov/pubmed/34050373 http://dx.doi.org/10.1007/s00277-021-04547-3 |