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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Zapisane w:
Opis bibliograficzny
Wydane w:Hepatology
Główni autorzy: 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.
Format: Artigo
Język:Inglês
Wydane: John Wiley and Sons Inc. 2019
Hasła przedmiotowe:
Original Articles
Dostęp online: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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