Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice von Rafi, Mohammad A, Rao, Han Zhi, Luzi, Paola, Wenger, David A

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Krabbe disease: One Hundred years from the bedside to the bench to the bedside von Wenger, David A., Rafi, Mohammad A., Luzi, Paola

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Consensus guidelines for newborn screening, diagnosis and treatment of infantile Krabbe disease von Kwon, Jennifer M, Matern, Dietrich, Kurtzberg, Joanne, Wrabetz, Lawrence, Gelb, Michael H, Wenger, David A, Ficicioglu, Can, Waldman, Amy T, Burton, Barbara K, Hopkins, Patrick V, Orsini, Joseph J

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Rare Saposin A deficiency: Novel variant and psychosine analysis von Calderwood, Laurel, Wenger, David A., Matern, Dietrich, Dahmoush, Hisham, Watiker, Valerie, Lee, Chung

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AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease) von Bradbury, Allison M, Rafi, Mohammed A, Bagel, Jessica H, Brisson, Becky K, Marshall, Michael S, Pesayco Salvador, Jill, Jiang, Xuntain, Swain, Gary P, Prociuk, Maria L, ODonnell, Patricia A, Fitzgerald, Caitlin, Ory, Daniel S, Bongarzone, Ernesto R, Shelton, G Diane, Wenger, David A, Vite, Charles H

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Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months von Beltran-Quintero, Maria L, Bascou, Nicholas A, Poe, Michele D, Wenger, David A, Saavedra-Matiz, Carlos A, Nichols, Matthew J, Escolar, Maria L

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Newborn screening for Krabbe disease in New York State: the first eight years’ experience von Orsini, Joseph J., Kay, Denise M., Saavedra-Matiz, Carlos A., Wenger, David A., Duffner, Patricia K., Erbe, Richard W., Biski, Chad, Martin, Monica, Krein, Lea M., Nichols, Matthew, Kurtzberg, Joanne, Escolar, Maria L., Adams, Darius J., Arnold, Georgianne L., Iglesias, Alejandro, Galvin-Parton, Patricia, Kronn, David F., Kwon, Jennifer M., Levy, Paul A., Pellegrino, Joan E., Shur, Natasha, Wasserstein, Melissa P., Caggana, Michele

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Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe Disease von Rafi, Mohammad A, Rao, Han Zhi, Luzi, Paola, Curtis, Mark T, Wenger, David A

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Pericardial Fat and Right Ventricular Morphology: The Multi-Ethnic Study of Atherosclerosis- Right Ventricle Study (MESA-RV) von Wenger, David S, Kawut, Steven M, Ding, Jingzhong, Bluemke, David A, Hough, Catherine L, Kronmal, Richard A, Lima, Joao A, Leary, Peter J

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Enzyme replacement therapy of a novel humanized mouse model of globoid cell leukodystrophy von Matthes, Frank, Andersson, Claes, Stein, Axel, Eistrup, Carl, Fogh, Jens, Gieselmann, Volkmar, Wenger, David A., Matzner, Ulrich

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Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease von Rafi, Mohammad A., Luzi, Paola, Wenger, David A.

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Clinical outcomes of children with abnormal newborn screening results for Krabbe disease in New York State von Wasserstein, Melissa P., Andriola, Mary, Arnold, Georgianne, Aron, Alan, Duffner, Patricia, Erbe, Richard W., Escolar, Maria L., Estrella, Lissette, Galvin-Parton, Patricia, Iglesias, Alejandro, Kay, Denise M., Kronn, David F., Kurtzberg, Joanne, Kwon, Jennifer M., Langan, Thomas J., Levy, Paul A., Naidich, Thomas P., Orsini, Joseph J., Pellegrino, Joan E., Provenzale, James M., Wenger, David A., Caggana, Michele

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Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT? von Rafi, Mohammad A, Luzi, Paola, Wenger, David A

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Advances in the Diagnosis and Treatment of Krabbe Disease von Wenger, David A, Luzi, Paola, Rafi, Mohammad A

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The Lysosomal Diseases Testing Laboratory: A review of the past 47 years von Wenger, David A., Luzi, Paola

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Transplantation of Umbilical-Cord Blood in Babies with Infantile Krabbe's Disease von Escolar, Maria L, Poe, Michele D, Provenzale, James M, Richards, Karen C, Allison, June, Wood, Susan, Wenger, David A, Pietryga, Daniel, Wall, Donna, Champagne, Martin, Morse, Richard, Krivit, William, Kurtzberg, Joanne

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Krabbe disease: Are certain mutations disease-causing only when specific polymorphisms are present or when inherited in trans with specific second mutations? von Wenger, David A., Luzi, Paola, Rafi, Mohammad A.

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Chemical mutagenesis of a GPCR ligand: Detoxifying “inflammo-attraction” to direct therapeutic stem cell migration von Lee, Jean-Pyo, Zhang, Runquan, Yan, Maocai, Duggineni, Srinivas, Wakeman, Dustin R., Niles, Walter L., Feng, Yongmei, Chen, Justin, Hamblin, Milton H., Hand, Edward B., Gonzalez, Rodolfo, Fang, Xiao, Zhu, Yinsong, Wang, Juan, Xu, Yan, Wenger, David A., Seyfried, Thomas N., An, Jing, Sidman, Richard L., Huang, Ziwei, Snyder, Evan Y.

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Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of... von Rafi, Mohammad A., Rao, Han Zhi, Luzi, Paola, Luddi, Alice, Curtis, Mark T., Wenger, David A.

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Lysosomal storage disease spectrum in nonimmune hydrops fetalis: a retrospective case control study von Al‐Kouatly, Huda B., Felder, Laura, Makhamreh, Mona M., Kass, Stephanie L., Vora, Neeta L., Berghella, Vincenzo, Berger, Seth, Wenger, David A., Luzi, Paola

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