Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach von Adang, Laura Ann, Sevagamoorthy, Anjana, Sherbini, Omar, Fraser, Jamie L., Bonkowsky, Joshua L., Gavazzi, Francesco, D'Aiello, Russel, Modesti, Nicholson B., Yu, Emily, Mutua, Sylvia, Kotes, Emma, Shults, Justine, Vincent, Ariel, Emrick, Lisa T., Keller, Stephanie, Van Haren, Keith P., Woidill, Sarah, Barcelos, Isabella, Pizzino, Amy, Schmidt, Johanna L., Eichler, Florian, Fatemi, Ali, Vanderver, Adeline

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Immunotherapy in Autoimmune Encephalitis: So Many Options, So Few Guidelines von Srivastava, Isha N, Van Haren, Keith P

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International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy: A Consensus-Based Approach von Engelen, Marc, van Ballegoij, Wouter J.C., Mallack, Eric James, Van Haren, Keith P., Köhler, Wolfgang, Salsano, Ettore, van Trotsenburg, A.S.P., Mochel, Fanny, Sevin, Caroline, Regelmann, Molly O., Tritos, Nicholas A., Halper, Alyssa, Lachmann, Robin H., Davison, James, Raymond, Gerald V., Lund, Troy C., Orchard, Paul J., Kuehl, Joern-Sven, Lindemans, Caroline A., Caruso, Paul, Turk, Bela Rui, Moser, Ann B., Vaz, Frédéric M., Ferdinandusse, Sacha, Kemp, Stephan, Fatemi, Ali, Eichler, Florian S., Huffnagel, Irene C.

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Presymptomatic Lesion in Childhood Cerebral Adrenoleukodystrophy: Timing and Treatment von Mallack, Eric James, Van Haren, Keith P., Torrey, Anna, van de Stadt, Stephanie, Engelen, Marc, Raymond, Gerald V., Fatemi, Ali, Eichler, Florian S.

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Pediatric Bickerstaff brainstem encephalitis: a systematic review of literature and case series von Santoro, Jonathan Douglas, Lazzareschi, Daniel V., Campen, Cynthia Jane, Van Haren, Keith P.

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A Novel Mouse Model for Cerebral Inflammatory Demyelination in X-Linked Adrenoleukodystrophy: Insights into Pathogenesis and Potential Therapeutic Targets von Hashemi, Ezzat, Srivastava, Isha N, Aguirre, Alejandro, Yoseph, Ezra T, Kaushal, Esha, Awani, Avni, Ryu, Jae K, Akassoglou, Katerina, Talebian, Shahrzad, Chu, Pauline, Pisani, Laura, Musolino, Patricia, Steinman, Lawrence, Doyle, Kristian, Robinson, William H, Sharpe, Orr, Cayrol, Romain, Orchard, Paul J, Lund, Troy, Vogel, Hannes, Lenail, Max, Han, May H, Bonkowsky, Joshua L, Van Haren, Keith P

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Vitamin D status and latitude predict brain lesions in adrenoleukodystrophy von van Haren, Keith P., Wilkes, Jacob, Moser, Ann B., Raymond, Gerald V., Richardson, Troy, Aubourg, Patrick, Collins, Timothy W., Mowry, Ellen M., Bonkowsky, Joshua L.

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A Phase 1 Study of Oral Vitamin D 3 in Boys and Young Men With X-Linked Adrenoleukodystrophy von Van Haren, Keith P, Cunanan, Kristen, Awani, Avni, Gu, Meng, Peña, Dalia, Chromik, Lindsay C, Považan, Michal, Rossi, Nicole C, Goodman, Jordan, Sundaram, Vandana, Winterbottom, Jennifer, Raymond, Gerald V, Cowan, Tina, Enns, Gregory M, Waubant, Emmanuelle, Steinman, Lawrence, Barker, Peter B, Spielman, Daniel, Fatemi, Ali

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A Phase 1 Study of Oral Vitamin D3 in Boys and Young Men With X-Linked Adrenoleukodystrophy von Van Haren, Keith P, Cunanan, Kristen, Awani, Avni, Gu, Meng, Peña, Dalia, Chromik, Lindsay C, Považan, Michal, Rossi, Nicole C, Goodman, Jordan, Sundaram, Vandana, Winterbottom, Jennifer, Raymond, Gerald V, Cowan, Tina, Enns, Gregory M, Waubant, Emmanuelle, Steinman, Lawrence, Barker, Peter B, Spielman, Daniel, Fatemi, Ali

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Presymptomatic Lesion in Childhood Cerebral Adrenoleukodystrophy: Timing and Treatment von Mallack, Eric James, Van Haren, Keith P., Torrey, Anna, van de Stadt, Stephanie, Engelen, Marc, Raymond, Gerald V., Fatemi, Ali, Eichler, Florian S.

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International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy von Engelen, Marc, van Ballegoij, Wouter J.C., Mallack, Eric James, van Haren, Keith P, Köhler, Wolfgang, Salsano, Ettore, van Trotsenburg, A.S.P., Mochel, Fanny, Sevin, Caroline, Regelmann, Molly O, Tritos, Nicholas A, Halper, Alyssa, Lachmann, Robin H, Davison, James, Raymond, Gerald V, Lund, Troy C, Orchard, Paul J, Kuehl, Joern-Sven, Lindemans, Caroline A, Caruso, Paul, Turk, Bela Rui, Moser, Ann B, Vaz, Frédéric M, Ferdinandusse, Sacha, Kemp, Stephan, Fatemi, Ali, Eichler, Florian S, Huffnagel, Irene C

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International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy: A Consensus-Based Approach von Engelen, Marc, van Ballegoij, Wouter J.C., Mallack, Eric James, Van Haren, Keith P., Köhler, Wolfgang, Salsano, Ettore, van Trotsenburg, A.S.P., Mochel, Fanny, Sevin, Caroline, Regelman, Molly O, Tritos, Nicholas A, Halper, Alyssa, Lachmann, Robin H, Davison, James, Raymond, Gerald V., Lund, Troy, Orchard, Paul J., Kuehl, Joern-Sven, Lindemans, Caroline A., Caruso, Paul, Turk, Bela Rui, Moser, Ann B., Vaz, Frederic M, Ferdinandusse, Sacha, Kemp, Stephan, Fatemi, Ali, Eichler, Florian S., Huffnagel, Irene C.

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Identification of naturally occurring fatty acids of the myelin sheath that resolve neuroinflammation von Ho, PP, Kanter, JL, Johnson, AM, Srinagesh, HK, Chang, EJ, Purdy, TM, van Haren, K, Wikoff, WR, Kind, T, Khademi, M, Matloff, LY, Narayana, S, Hur, EM, Lindstrom, TM, He, ZG, Fiehn, O, Olsson, T, Han, XL, Han, MH, Steinman, L, Robinson, WH

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Clinical approach to the diagnosis of autoimmune encephalitis in the pediatric patient von Cellucci, Tania, Van Mater, Heather, Graus, Francesc, Muscal, Eyal, Gallentine, William, Klein-Gitelman, Marisa S., Benseler, Susanne M., Frankovich, Jennifer, Gorman, Mark P., Van Haren, Keith, Dalmau, Josep, Dale, Russell C.

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A novel outbreak enterovirus D68 strain associated with acute flaccid myelitis cases in the USA (2012–14): a retrospective cohort study von Greninger, Alexander L, MD, Naccache, Samia N, PhD, Messacar, Kevin, MD, Clayton, Anna, BSc, Yu, Guixia, BSc, Somasekar, Sneha, BSc, Federman, Scot, BA, Stryke, Doug, BSc, Anderson, Christopher, BSc, Yagi, Shigeo, PhD, Messenger, Sharon, PhD, Wadford, Debra, PhD, Xia, Dongxiang, MD, Watt, James P, MD, Van Haren, Keith, MD, Dominguez, Samuel R, MD, Glaser, Carol, MD, Aldrovandi, Grace, Prof, Chiu, Charles Y, Dr

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Acute Flaccid Myelitis of Unknown Etiology in California, 2012-2015 von Van Haren, Keith, Ayscue, Patrick, Waubant, Emmanuelle, Clayton, Anna, Sheriff, Heather, Yagi, Shigeo, Glenn-Finer, Rose, Padilla, Tasha, Strober, Jonathan B, Aldrovandi, Grace, Wadford, Debra A, Chiu, Charles Y, Xia, Dongxiang, Harriman, Kathleen, Watt, James P, Glaser, Carol A

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Allogeneic HSCT for adult-onset leukoencephalopathy with spheroids and pigmented glia von Gelfand, Jeffrey M, Greenfield, Ariele L, Barkovich, Matthew, Mendelsohn, Bryce A, Van Haren, Keith, Hess, Christopher P, Mannis, Gabriel N

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Genetic and phenotypic spectrum associated with IFIH1 gain‐of‐function von Rice, Gillian I., Park, Sehoon, Gavazzi, Francesco, Adang, Laura A., Ayuk, Loveline A., Van Eyck, Lien, Seabra, Luis, Barrea, Christophe, Battini, Roberta, Belot, Alexandre, Berg, Stefan, Billette de Villemeur, Thierry, Bley, Annette E., Blumkin, Lubov, Boespflug‐Tanguy, Odile, Briggs, Tracy A., Brimble, Elise, Dale, Russell C., Darin, Niklas, Debray, François‐Guillaume, De Giorgis, Valentina, Denecke, Jonas, Doummar, Diane, Drake af Hagelsrum, Gunilla, Eleftheriou, Despina, Estienne, Margherita, Fazzi, Elisa, Feillet, François, Galli, Jessica, Hartog, Nicholas, Harvengt, Julie, Heron, Bénédicte, Heron, Delphine, Kelly, Diedre A., Lev, Dorit, Levrat, Virginie, Livingston, John H., Marti, Itxaso, Mignot, Cyril, Mochel, Fanny, Nougues, Marie‐Christine, Oppermann, Ilena, Pérez‐Dueñas, Belén, Popp, Bernt, Rodero, Mathieu P., Rodriguez, Diana, Saletti, Veronica, Sharpe, Cia, Tonduti, Davide, Vadlamani, Gayatri, Van Haren, Keith, Tomas Vila, Miguel, Vogt, Julie, Wassmer, Evangeline, Wiedemann, Arnaud, Wilson, Callum J., Zerem, Ayelet, Zweier, Christiane, Zuberi, Sameer M., Orcesi, Simona, Vanderver, Adeline L., Hur, Sun, Crow, Yanick J.

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The Unfolded Protein Response in Vanishing White Matter Disease von van der Voorn, J Patrick, van Kollenburg, Barbara, Bertrand, Gesina, Van Haren, Keith, Scheper, Gert C, Powers, James M, van der Knaap, Marjo S

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Safety, tolerability, and efficacy of fluoxetine as an antiviral for acute flaccid myelitis von Messacar, Kevin, Sillau, Stefan, Hopkins, Sarah E, Otten, Catherine, Wilson-Murphy, Molly, Wong, Brian, Santoro, Jonathan D, Treister, Andrew, Bains, Harlori K, Torres, Alcy, Zabrocki, Luke, Glanternik, Julia R, Hurst, Amanda L, Martin, Jan A, Schreiner, Teri, Makhani, Naila, DeBiasi, Roberta L, Kruer, Michael C, Tremoulet, Adriana H, Van Haren, Keith, Desai, Jay, Benson, Leslie A, Gorman, Mark P, Abzug, Mark J, Tyler, Kenneth L, Dominguez, Samuel R

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