Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III von Ghosh, Arunabha, Shapiro, Elsa, Rust, Stewart, Delaney, Kathleen, Parker, Samantha, Shaywitz, Adam J, Morte, Adelaida, Bubb, Gillian, Cleary, Maureen, Bo, Tien, Lavery, Christine, Bigger, Brian W, Jones, Simon A

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O08: Long-term efficacy and safety of pegzilarginase for arginase 1 deficiency: 2 years of experience in the phase 2 extension study von McNutt, Markey, Diaz, George, Schulze, Andreas, Bubb, Gillian, Sloan, Leslie, Leão-Teles, Elisa, Batzios, Spyros, Sun, Angela, Bannick, Allison, Zori, Roberto, Enns, Gregory

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Natural history of arginase 1 deficiency and the unmet needs of patients: A systematic review of case reports von Bin Sawad, Aseel, Pothukuchy, Arti, Badeaux, Mark, Hodson, Victoria, Bubb, Gillian, Lindsley, Kristina, Uyei, Jennifer, Diaz, George A.

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PEGZILARGINASE IN ARGINASE 1 DEFICIENCY: RESULTS OF THE PEACE PIVOTAL PHASE 3 CLINICAL TRIAL von Enns, Gregory M., Russo, Rossana Sanchez, Bradford, Eric, Bubb, Gillian, Sloan, Leslie S., Gasperini, Serena, Diaz, George A.

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Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE): a phase 3, randomized, double-blind, placebo-controlled, multi-centre trialResearch in context von Rossana Sanchez Russo, Serena Gasperini, Gillian Bubb, Linda Neuman, Leslie S. Sloan, George A. Diaz, Gregory M. Enns

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Clinical effect and safety profile of pegzilarginase in patients with arginase 1 deficiency von Diaz, George A., Schulze, Andreas, McNutt, Markey C., Leão‐Teles, Elisa, Merritt, J. Lawrence, Enns, Gregory M., Batzios, Spyros, Bannick, Allison, Zori, Roberto T., Sloan, Leslie S., Potts, Susan L., Bubb, Gillian, Quinn, Anthony G.

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Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE): a phase 3, randomized, double-blind, placebo-controlled, multi-centre trial von Russo, Rossana Sanchez, Gasperini, Serena, Bubb, Gillian, Neuman, Linda, Sloan, Leslie S., Diaz, George A., Enns, Gregory M.

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Final results of the phase 1/2, open-label clinical study of intravenous recombinant human N-acetyl-α-d-glucosaminidase (SBC-103) in children with mucopolysaccharidosis IIIB von Whitley, Chester B., Vijay, Suresh, Yao, Bert, Pineda, Mercé, Parker, Geoff J.M., Rojas-Caro, Sandra, Zhang, Xiaoping, Dai, Yang, Cinar, Amy, Bubb, Gillian, Patki, Kiran C., Escolar, Maria L.

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Novel NAGLU variants associated with MPS IIIB and in vitro analysis of residual enzymatic activities von Ji, Rui-Ru, Whitley, Chester B., Escolar, Maria L., Vijay, Suresh, Wijburg, Frits A., Giugliani, Roberto, Hutchinson, Andrew, Jain, Nina, Forbes, Chris, Bubb, Gillian, Patki, Kiran C.

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Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III von Ghosh, Arunabha, Shapiro, Elsa, Rust, Stewart, Delaney, Kathleen, Parker, Samantha, Shaywitz, Adam J, Morte, Adelaida, Bubb, Gillian, Cleary, Maureen, Bo, Tien, Lavery, Christine, Bigger, Brian, Jones, Simon

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