Osseous Manifestations of Adult Gaucher Disease in the Era of Enzyme Replacement Therapy von Deegan, Patrick B., Pavlova, Elena, Tindall, Jane, Stein, Penelope E., Bearcroft, Philip, Mehta, Atul, Hughes, Derralynn, Wraith, J. Edmund, Cox, Timothy M.

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Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease von Nicolino, Marc, Byrne, Barry, Wraith, J Edmund, Leslie, Nancy, Mandel, Hanna, Freyer, David R, Arnold, Georgianne L, Pivnick, Eniko K, Ottinger, C J, Robinson, Peter H, Loo, John-Charles A, Smitka, Martin, Jardine, Philip, Tatò, Luciano, Chabrol, Brigitte, McCandless, Shawn, Kimura, Shigemi, Mehta, L, Bali, Deeksha, Skrinar, Alison, Morgan, Claire, Rangachari, Lakshmi, Corzo, Deya, Kishnani, Priya S

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Homozygous loss of a cysteine residue in the glucocerebrosidase gene results in Gaucher's disease with a hydropic phenotype von Church, Heather J, Cooper, Alan, Stewart, Fiona, Thornton, Claire M, Wraith, J Edmund

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