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Maralixibat for the treatment of PFIC: Long‐term, IBAT inhibition in an open‐label, Phase 2 study

Children with progressive familial intrahepatic cholestasis, including bile salt export pump (BSEP) and familial intrahepatic cholestasis–associated protein 1 (FIC1) deficiencies, suffer debilitating cholestatic pruritus that adversely affects growth and quality of life (QoL). Reliance on surgical i...

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Detalles Bibliográficos
Autores principales: Loomes, Kathleen M., Squires, Robert H., Kelly, Deirdre, Rajwal, Sanjay, Soufi, Nisreen, Lachaux, Alain, Jankowska, Irena, Mack, Cara, Setchell, Kenneth D. R., Karthikeyan, Palaniswamy, Kennedy, Ciara, Dorenbaum, Alejandro, Desai, Nirav K., Garner, Will, Jaecklin, Thomas, Vig, Pamela, Miethke, Alexander, Thompson, Richard J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9426380/
https://www.ncbi.nlm.nih.gov/pubmed/35507739
http://dx.doi.org/10.1002/hep4.1980