Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiencyMolecular Genetics and Metabolism - Tập 116 - Trang 88-97 - 2015
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