Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency

Melissa P. Wasserstein, Simon A. Jones, Handrean Soran, George A. Diaz, Natalie Lippa, Beth L. Thurberg, Kerry Culm-Merdek, Elias Shamiyeh, Haig Inguilizian, Gerald F. Cox, Ana Cristina Puga

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38 Scopus citations

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Chemical Compounds

Medicine & Life Sciences