Adults with chronic acid sphingomyelinase deficiency show significant visceral, pulmonary, and hematologic improvements after enzyme replacement therapy with olipudase-alfa: 1-year results of the ASCEND placebo-controlled trial

MOLECULAR GENETICS AND METABOLISM

卷 132, 期 2, 页码 S110-S110

ACADEMIC PRESS INC ELSEVIER SCIENCE

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Endocrinology & Metabolism Genetics & Heredity Medicine, Research & Experimental