Hematopoietic Stem-Cell Transplantation in Globoid-Cell Leukodystrophy von Krivit, William, Shapiro, Elsa G, Peters, Charles, Wagner, John E, Cornu, Guy, Kurtzberg, Joanne, Wenger, David A, Kolodny, Edwin H, Vanier, Marie T, Loes, Daniel J, Dusenbery, Kathryn, Lockman, Lawrence A

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Sphingomyelinase Deficiency (Niemann-Pick disease) in a Hereford Calf von Saunders, G.K, Wenger, D.A

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GM2 Gangliosidosis in Shiba Inu Dogs with an In‐Frame Deletion in HEXB von Kolicheski, A., Johnson, G.S., Villani, N.A., O'Brien, D.P., Mhlanga‐Mutangadura, T., Wenger, D.A., Mikoloski, K., Eagleson, J.S., Taylor, J.F., Schnabel, R.D., Katz, M.L.

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Abnormal neuronal metabolism and storage in mucopolysaccharidosis type VI (Maroteaux-Lamy) disease von Walkley, S. U., Thrall, M. A., Haskins, M. E., Mitchell, T. W., Wenger, D. A., Brown, D. E., Dial, S., Seim, H.

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Addressing multiple problems in the family practice office visit von Flocke, S A, Frank, S H, Wenger, D A

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Sphingomyelin Lipidosis (Niemann–Pick Disease) in a Juvenile Raccoon (Procyon lotor) von Vapniarsky, N., Wenger, D.A., Scheenstra, D., Mete, A.

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Improved outcomes for hospitalized asthmatic children using a clinical pathway von Kelly, Cynthia Szelc, Andersen, Charlotte Littelman, Pestian, John P., Wenger, Andrea D., Finch, Albert B., Strope, Gerald L., Luckstead, Eugene F.

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MR Imaging and Proton MR Spectroscopy in Adult Krabbe Disease von Farina, Laura, Bizzi, Alberto, Finocchiaro, Gaetano, Pareyson, Davide, Sghirlanzoni, Angelo, Bertagnolio, Barbara, and Savoiardo, Mario, Naidu, and SakkuBai, Singhal, Bhim S, Wenger, David A

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Engraftment following in utero bone marrow transplantation for globoid cell leukodystrophy von BAMBACH, B. J, MOSER, H. W, BLAKEMORE, K, CORSON, V. L, GRIFFIN, C. A, NOGA, S. J, PERLMAN, E. J, ZUCKERMAN, R, WENGER, D. A, JONES, R. J

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Retrovirus-Mediated Gene Transfer and Galactocerebrosidase Uptake into Twitcher Glial Cells Results in Appropriate Localization and Phenotype Correction von Luddi, A., Volterrani, M., Strazza, M., Smorlesi, A., Rafi, M.A., Datto, J., Wenger, D.A., Costantino-Ceccarini, E.

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Effects of dietary cholesterol restriction in a feline model of Niemann–Pick type C disease von Somers, K. L., Brown, D. E., Fulton, R., Schultheiss, P. C., Hamar, D., Smith, M. O., Allison, R., Connally, H. E., Just, C., Mitchell, T. W., Wenger, D. A., Thrall, M. A.

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Clinical course and biochemistry of sialuria von Enns, G. M., Seppala, R., Musci, T. J., Weisiger, K., Ferrell, L. D., Wenger, D. A., Gahl, W. A., Packman, S.

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Cloning and expression cDNA encoding human galactocerebrosidase, the enzyme deficient in globoid cell leukodystrophy von Chen, Yue Qun, Rafi, Mohammad A., de Gala, Gregory, Wenger, David A.

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A defect in cholesterol esterification in Niemann-Pick disease (type C) patients von Pentchev, Peter G., Comly, Marcella E., Kruth, Howard S., Vanier, Marie T., Wenger, David A., Patel, Shutish, Brady, Roscoe O.

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Niemann-Pick C1 Disease: The I1061T Substitution Is a Frequent Mutant Allele in Patients of Western European Descent and Correlates with a Classic Juvenile Phenotype von Millat, Gilles, Marçais, Christophe, Rafi, Mohammad A., Yamamoto, Toshiyuki, Morris, Jill A., Pentchev, Peter G., Ohno, Kousaku, Wenger, David A., Vanier, Marie T.

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A large deletion together with a point mutation in the GALC gene is a common mutant allele in patients with infantile Krabbe disease von Rafi, Mohammad A., Luzi, Paola, Chen, Yue Qun, Wenger, David A.

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Characterization of the large deletion in the GALC gene found in patients with Krabbe disease von Luzi, Paola, Rafi, Mohammad A., Wenger, David A.

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Niemann-Pick C1 Disease: Correlations between NPC1 Mutations, Levels of NPC1 Protein, and Phenotypes Emphasize the Functional Significance of the Putative Sterol-Sensing Domain and... von Millat, Gilles, Marçais, Christophe, Tomasetto, Catherine, Chikh, Karim, Fensom, Anthony H., Harzer, Klaus, Wenger, David A., Ohno, K., Vanier, Marie T.

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Globoid cell leukodystrophy in Cairn and West Highland white terriers von Wenger, D.A, Victoria, T, Rafi, M.A, Luzi, P, Vanier, M.T, Vite, C, Patterson, D.F, Haskins, M.H

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Characterization of the Rhesus Monkey Galactocerebrosidase (GALC) cDNA and Gene and Identification of the Mutation Causing Globoid Cell Leukodystrophy (Krabbe Disease) in This Prim... von Luzi, Paola, Rafi, Mohammad A., Victoria, Teresa, Baskin, Gary B., Wenger, David A.

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