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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2019
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7255459/
https://www.ncbi.nlm.nih.gov/pubmed/31512765
http://dx.doi.org/10.1002/hep.30936

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