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...

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Detalles Bibliográficos
Autores principales: Petrides, Petro E., Klein, Michael, Schuhmann, Elfriede, Torkler, Heike, Molitor, Brigitte, Loehr, Christian, Obermeier, Zahra, Beykirch, Maria K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2021
Materias:
Original Article
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

Internet

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

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