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Overlapping and divergent hepatic and lipoprotein phenotypes in untreated adults with acid sphingomyelinase deficiency versus untreated adults with Gaucher disease from two pivotal clinical trialsCassiman, David ; Mistry, Pramod K ; Jones, Simon A ; Lachmann, Robin ; Lukina, Elena ; Prada, Carlos E ; Thurberg, Beth L ; Wasserstein, Melissa P ; Foster, Meredith C ; Patel, Reema M ; Ribes, Manuel ; Underhill, Lisa H ; Peterschmitt, M. JudithMOLECULAR GENETICS AND METABOLISM, 2022, Vol.135 (2), p.S29-S29ACADEMIC PRESS INC ELSEVIER SCIENCETexto completo disponível |