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Publications

Kang, H-R, Waskowicz, L, Seifts, AM, Landau, DJ, Young, SP, and Koeberl, DD. "Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia.(Accepted)" Molecular Therapy. Methods & Clinical Development 13 (June 2019): 265-273.

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Kang, H-R, Waskowicz, L, Seifts, AM, Landau, DJ, Young, SP, and Koeberl, DD. "Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia.(Accepted)" Molecular Therapy. Methods & Clinical Development 13 (June 2019): 265-273.

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Shulman, DS, Mehrotra, P, Blonquist, TM, Capraro, A, Lehmann, LE, Silverman, LB, Surana, NK, and Place, AE. "A single institutional review of pediatric Bacillus spp. bloodstream infections demonstrates increased incidence among children with cancer.(Accepted)" Pediatric Blood & Cancer 66, no. 4 (April 2019): e27568-null.

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Krishnamurti, L, Neuberg, DS, Sullivan, KM, Kamani, NR, Abraham, A, Campigotto, F, Zhang, W, Dahdoul, T, De Castro, L, Parikh, S, Bakshi, N, Haight, A, Hassell, KL, Loving, R, Rosenthal, J, Smith, SL, Smith, W, Spearman, M, Stevenson, K, Wu, CJ, Wiedl, C, Waller, EK, and Walters, MC. "Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study.(Accepted)" American Journal of Hematology 94, no. 4 (April 2019): 446-454.

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Goldstein, RF, and Malcolm, WF. "Care of the Neonatal Intensive Care Unit Graduate after Discharge.(Accepted)" Pediatric Clinics of North America 66, no. 2 (April 2019): 489-508. (Review)

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Parikh, SH, Pentz, RD, Haight, A, Adeli, M, Martin, PL, Driscoll, TA, Page, K, Kurtzberg, J, Prasad, VK, and Barfield, RC. "Ethical considerations of using a single minor donor for three bone marrow harvests for three HLA-matched siblings with primary immunodeficiency.(Accepted)" Pediatric Blood & Cancer 66, no. 4 (April 2019): e27602-null.

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Kishnani, PS, Rockman-Greenberg, C, Rauch, F, Bhatti, MT, Moseley, S, Denker, AE, Watsky, E, and Whyte, MP. "Five-year efficacy and safety of asfotase alfa therapy for adults and adolescents with hypophosphatasia.(Accepted)" Bone 121 (April 2019): 149-162.

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Balevic, SJ, Green, TP, Clowse, MEB, Eudy, AM, Schanberg, LE, and Cohen-Wolkowiez, M. "Pharmacokinetics of Hydroxychloroquine in Pregnancies with Rheumatic Diseases.(Accepted)" Clinical Pharmacokinetics 58, no. 4 (April 2019): 525-533.

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Lim, JA, Yi, H, Gao, F, Raben, N, Kishnani, PS, and Sun, B. "Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease(Accepted)." Molecular Therapy Methods and Clinical Development 12 (March 15, 2019): 233-245.

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Helbig, KL, Lauerer, RJ, Bahr, JC, Souza, IA, Myers, CT, Uysal, B, Schwarz, N, Gandini, MA, Huang, S, Keren, B, Mignot, C, Afenjar, A, Billette de Villemeur, T, Héron, D, Nava, C, Valence, S, Buratti, J, Fagerberg, CR, Soerensen, KP, Kibaek, M, Kamsteeg, EJ, Koolen, DA, Gunning, B, Schelhaas, HJ, Kruer, MC, Fox, J, Bakhtiari, S, Jarrar, R, Padilla-Lopez, S, Lindstrom, K, Jin, SC, Zeng, X, Bilguvar, K, Papavasileiou, A, Xing, Q, Zhu, C, Boysen, K, Vairo, F, Lanpher, BC, Klee, EW, Tillema, JM, Payne, ET, Cousin, MA, Kruisselbrink, TM, Wick, MJ, Baker, J, Haan, E, Smith, N, Sadeghpour, A, Davis, EE, Katsanis, N, Allori, A, Angrist, M, Ashley, P, Bidegain, M, Boyd, B, Chambers, E, Cope, H, Cotten, CM, Curington, T, Ellestad, S, Fisher, K, French, A, Gallentine, W, Goldberg, R, Hill, K, Kansagra, S, Katsanis, S, Kurtzberg, J, Marcus, J, McDonald, M, Mikati, M, Miller, S, Murtha, A, Perilla, Y, Pizoli, C, Purves, T, Ross, S, Smith, E, Wiener, J, Corbett, MA, MacLennan, AH, Gecz, J, Biskup, S, Goldmann, E, Rodan, LH, Kichula, E, Segal, E, Jackson, KE, Asamoah, A, Dimmock, D, McCarrier, J, Botto, LD, Filloux, F, Tvrdik, T, Cascino, GD, and Klingerman, S. "Erratum: De Novo Pathogenic Variants in CACNA1E Cause Developmental and Epileptic Encephalopathy with Contractures, Macrocephaly, and Dyskinesias (The American Journal of Human Genetics (2018) 103(5) (666–678), (S0002929718303173), (10.1016/j.ajhg.2018.09.006))." American Journal of Human Genetics 104, no. 3 (March 7, 2019): 562-null.

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