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Goldin E, Zheng W, Motabar O, Southall N, Choi JH, Marugan J, Austin CP, Sidransky E(2012) High throughput screening for small molecule therapy for Gaucher disease usingpatient tissue as the source of mutant glucocerebrosidase. Muenzer J, Beck M, Eng CM, Giugliani R, Harmatz P, Martin R, Ramaswami U, Vellodi A,Wraith JE, Cleary M, Gucsavas-Calikoglu M, Puga AC, Shinawi M, Ulbrich B, VijayaraghavanS, Wendt S, Conway AM, Rossi A, Whiteman DAH, Kimura A (2011) Long-term, open- labeledextension study of idursulfase in the treatment of Hunter syndrome

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