Miglustat in adult and juvenile patients with Niemann–Pick disease type C: Long-term data from a clinical trial

J. E. Wraith, Darleen Vecchio, Elizabeth Jacklin, Larry A. Abel, Harbajan Chadha‐Boreham, Cecile Paquet Luzy, R. Giorgino, Marc C. Patterson

Molecular Genetics and Metabolism · 2009 · 210 citations · 23 references

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