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EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks

BACKGROUND AND AIMS: Acute hepatic porphyria comprises a group of rare genetic diseases caused by mutations in genes involved in heme biosynthesis. Patients can experience acute neurovisceral attacks, debilitating chronic symptoms, and long‐term complications. There is a lack of multinational, prosp...

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Detalhes bibliográficos
Publicado no:Hepatology
Main Authors: Gouya, Laurent, Ventura, Paolo, Balwani, Manisha, Bissell, D. Montgomery, Rees, David C., Stölzel, Ulrich, Phillips, John D., Kauppinen, Raili, Langendonk, Janneke G., Desnick, Robert J., Deybach, Jean‐Charles, Bonkovsky, Herbert L., Parker, Charles, Naik, Hetanshi, Badminton, Michael, Stein, Penelope E., Minder, Elisabeth, Windyga, Jerzy, Bruha, Radan, Cappellini, Maria Domenica, Sardh, Eliane, Harper, Pauline, Sandberg, Sverre, Aarsand, Aasne K., Andersen, Janice, Alegre, Félix, Ivanova, Aneta, Talbi, Neila, Chan, Amy, Querbes, William, Ko, John, Penz, Craig, Liu, Shangbin, Lin, Tim, Simon, Amy, Anderson, Karl E.
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2019
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7255459/
https://ncbi.nlm.nih.gov/pubmed/31512765
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/hep.30936
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