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Hepatoerythropoietic porphyria in a 2-year-old child: Clinical features and effects of treatment 2岁儿童肝卟啉症的临床特征及治疗效果 Bensaber, Narmene; Tran, Benny; Rebeiz, Lina; Wang, Chenqing; Tortorelli, Silvia; Yalom, Lenore K.; Trinidad, John; Wiltsie, Laura; Kerkar, Nanda; Moghe, Akshata; Anderson, Karl E.; Leaf, Rebecca K.; Yeung, Amy K. 分享 收藏
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Liver involvement in a large cohort of patients with erythropoietic protoporphyria or X-linked protoporphyria Levy, Cynthia; Naik, Hetanshi; Overbey, Jessica; Hedstrom, Karli; Wang, Kelly; Mcdonough, Catherine; Freeman, Mary; Keel, Sioban B.; Erwin, Angelika L.; Dickey, Amy K.; Leaf, Rebecca K.; Quigley, John; Mazepa, Marshall; Wang, Bruce; Phillips, John; Parker, Charles; Mcguire, Brendan; Kazamel, Mohamed; Bonkovsky, Herbert; Rudnick, Sean; Anderson, Karl E.; Moghe, Akshata; Thapar, Manish; Saberi, Behnam; Wheeden, Kristen; Desnick, Robert; Balwani, Manisha; Porphyrias Consortium of the Rare Diseases Clinical Res Network 分享 收藏
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Lipid-encapsulated mRNA encoding an extended serum half-life interleukin-22 ameliorates metabolic disease in mice Canali, Susanna; Fischer, Alexander W.; Nguyen, Mychael; Anderson, Karl; Wu, Lorna; Graham, Anne-Renee; Hsiao, Chiaowen Joyce; Bankar, Chinmayi; Dussault, Nancy; Ritchie, Veronica; Goodridge, Meagan; Sparrow, Todd; Pannoni, Allison; Tse, Sze-Wah; Woo, Vivienne; Klovdahl, Kaitlin; Iacovelli, Jared; Huang, Eric 分享 收藏
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Afamelanotide for Treatment of the Protoporphyrias: Impact on Quality of Life and Laboratory Parameters in a US Cohort Leaf, Rebecca K.; Naik, Hetanshi; Jiang, Paul Y.; Elmariah, Sarina B.; Hodges, Pamela; Mead, Jennifer; Trinidad, John; Saberi, Behnam; Tran, Benny; Valiante, Sarah; Mernick, Francesca; Leaf, David E.; Anderson, Karl E.; Dickey, Amy K. 分享 收藏
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Evidence-based consensus guidelines for the diagnosis and management of protoporphyria-related liver dysfunction in erythropoietic protoporphyria and X-linked protoporphyria Levy, Cynthia; Dickey, Amy K.; Wang, Bruce; Thapar, Manish; Naik, Hetanshi; Keel, Sioban B.; Saberi, Behnam; Beaven, Simon W.; Rudnick, Sean R.; Elmariah, Sarina B.; Erwin, Angelika L.; Goddu, Robert J.; Hedstrom, Karli; Leaf, Rebecca Karp; Kazamel, Mohamed; Mazepa, Marshall; Philpotts, Lisa Liang; Quigley, John; Raef, Haya; Ungar, Jonathan; Anderson, Karl E.; Balwani, Manisha 分享 收藏
Prospective observational pilot study of quantitative light dosimetry in erythropoietic protoporphyria Dickey, Amy K.; Rebeiz, Lina; Raef, Haya; Leaf, Rebecca K.; Elmariah, Sarina; Naik, Hetanshi; Anderson, Karl; Conley, Jared; Iyasere, Christiana; Zhao, Sophia; Birkenfeld, Judith S.; Arroyo-Gallego, Teresa; Wheeden, Kristen; Ducamp, Sarah; Christiani, David C.; Fleming, Mark D.; Kochevar, Irene 分享 收藏