Gaucher disease: Progress and ongoing challenges von Mistry, Pramod K, Lopez, Grisel, Schiffmann, Raphael, Barton, Norman W, Weinreb, Neal J, Sidransky, Ellen

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Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double‐blind, multinational, Phase 3 study von Gonzalez, Derlis E., Turkia, Hadhami Ben, Lukina, Elena A., Kisinovsky, Isaac, Dridi, Marie‐Françoise Ben, Elstein, Deborah, Zahrieh, David, Crombez, Eric, Bhirangi, Kiran, Barton, Norman W., Zimran, Ari

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Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease von Turkia, Hadhami Ben, Gonzalez, Derlis E., Barton, Norman W., Zimran, Ari, Kabra, Madhulika, Lukina, Elena A., Giraldo, Pilar, Kisinovsky, Isaac, Bavdekar, Ashish, Dridi, Marie‐Françoise Ben, Gupta, Neerja, Kishnani, Priya S., Sureshkumar, E.K., Wang, Nan, Crombez, Eric, Bhirangi, Kiran, Mehta, Atul

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Roscoe Owen Brady, MD: Remembrances of co-investigators and colleagues von Desnick, Robert J, Barton, Norman W, Furbish, Scott, Grabowski, Gregory A, Karlsson, Stefan, Kolodny, Edwin H, Medin, Jeffrey A, Murray, Gary J, Mistry, Pramod K, Patterson, Marc C, Schiffmann, Raphael, Weinreb, Neal J

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The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher’s disease von Altarescu, Gheona, Hill, Suvimol, Wiggs, Edyth, Jeffries, Neal, Kreps, Constance, Parker, Colette C., Brady, Roscoe O., Barton, Norman W., Schiffmann, Raphael

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Sulfatide levels correlate with severity of neuropathy in metachromatic leukodystrophy von Dali, Christine í, Barton, Norman W., Farah, Mohamed H., Moldovan, Mihai, Månsson, Jan‐Eric, Nair, Nitin, Dunø, Morten, Risom, Lotte, Cao, Hongmei, Pan, Luying, Sellos‐Moura, Marcia, Corse, Andrea M., Krarup, Christian

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Physiological characterization of neuropathy in Fabry's disease von Luciano, Carlos A., Russell, James W., Banerjee, Tapas K., Quirk, Jane M., Scott, Leland J.C., Dambrosia, James M., Barton, Norman W., Schiffmann, Raphael

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Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's Disease von Barton, Norman W, Brady, Roscoe O, Dambrosia, James M, Di Bisceglie, Adrian M, Doppelt, Samuel H, Hill, Suvimol C, Mankin, Henry J, Murray, Gary J, Parker, Robert I, Argoff, Charles E, Grewal, Raji P, Yu, Kian-Ti

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Evaluation of recombinant human IGF-1/IGFBP-3 on intraventricular hemorrhage prevention and survival in the preterm rabbit pup model von Ekström, Claes, Ortenlöf, Niklas, Kristiansson, Amanda, Holmqvist, Bo, Jungner, Åsa, Vallius, Suvi, Wang, Xiaoyang, Hellström, Ann, Barton, Norman, Carey, Galen, Ley, David, Gram, Magnus

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Decreased Bone Density in Splenectomized Gaucher Patients Receiving Enzyme Replacement Therapy von Schiffmann, Raphael, Mankin, Henry, Dambrosia, James M, Xavier, Ramnik J, Kreps, Constance, Hill, Suvimol C, Barton, Norman W, Rosenthal, Daniel I

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Enzyme replacement therapy for Gaucher disease: skeletal responses to macrophage-targeted glucocerebrosidase von Rosenthal, D I, Doppelt, S H, Mankin, H J, Dambrosia, J M, Xavier, R J, McKusick, K A, Rosen, B R, Baker, J, Niklason, L T, Hill, S C

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Constitutive Achlorhydria in Mucolipidosis Type IV von Schiffmann, Raphael, Dwyer, Nancy K., Lubensky, Irina A., Tsokos, Maria, Sutliff, Vincent E., Latimer, John S., Frei, Karen P., Brady, Roscoe O., Barton, Norman W., Blanchette-Mackie, E. Joan, Goldin, Ehud

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Development and verification of a pharmacokinetic model to optimize physiologic replacement of rhIGF-1/rhIGFBP-3 in preterm infants von Chung, Jou-Ku, Hallberg, Boubou, Hansen-Pupp, Ingrid, Graham, Martin A., Fetterly, Gerald, Sharma, Jyoti, Tocoian, Adina, Kreher, Nerissa C., Barton, Norman, Hellström, Ann, Ley, David

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Management of Neutralizing Antibody to Ceredase in a Patient With Type 3 Gaucher Disease von Brady, Roscoe O, Murray, Gary J, Oliver, Katherine L, Leitman, Susan F, Sneller, Michael C, Fleisher, Thomas A, Barton, Norman W, Collaborators

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Treatment of Gaucher's Disease von Moscicki, Richard A, Taunton-Rigby, Alison, Barton, Norman W, Brady, Roscoe O, Dambrosia, James M, Grabowski, Gregory A, Hollak, Carla E.M, Aerts, J.M.F.G, van Oers, M.H.J, Sidransky, Ellen, Martin, Brian, Ginns, Edward I, Freiwald, David Ronald, Lexchin, Joel, Beutler, Ernest, Figueroa, Michael, Koziol, James, Garber, Alan M

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Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation von Dunbar, C E, Kohn, D B, Schiffmann, R, Barton, N W, Nolta, J A, Esplin, J A, Pensiero, M, Long, Z, Lockey, C, Emmons, R V, Csik, S, Leitman, S, Krebs, C B, Carter, C, Brady, R O, Karlsson, S

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Retroviral Transfer of the Glucocerebrosidase Gene into CD34 + Cells from Patients with Gaucher Disease: In Vivo Detection of Transduced Cells without Myeloablation von Dunbar, Cynthia E., Kohn, Donald B., Schiffmann, Raphael, Barton, Norman W., Nolta, Jan A., Esplin, Joan A., Pensiero, Michael, Long, Zhifeng, Lockey, Chris, Emmons, Robert V. B., Csik, Susie, Leitman, Susan, Krebs, Connie B., Carter, Charles, Brady, Roscoe O., Karlsson, Stefan

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Risk factors for osteonecrosis in patients with Type 1 Gaucher's disease von RODRIGUE, S. W, ROSENTHAL, D. I, BARTON, N. W, ZURAKOWSKI, D, MANKIN, H. J

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Therapeutic Response to Intravenous Infusions of Glucocerebrosidase in a Patient with Gaucher Disease von Barton, Norman W., Furbish, F. Scott, Murray, Gary J., Garfield, Mark, Brady, Roscoe O.

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Prospective study of neurological responses to treatment with macrophage-targeted glucocerebrosidase in patients with type 3 Gaucher's disease von Schiffmann, Raphael, Heyes, Melvyn P., Aerts, J. M., Dambrosia, James M., Patterson, Marc C., DeGraba, Thomas, Parker, Colette C., Zirzow, Gregory C., Oliver, Katherine, Tedeschi, Gioacchino, Brady, Roscoe O., Barton, Norman W.

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