Olipudase alfa for acid sphingomyelinase deficiency: Evidence of efficacy from a compassionate use program.
Gragnaniello V, Tagi VM, Carubbi F, Barbato A, Sibilio M, Fecarotta S, Pappalardo MG, Motta I, Sechi A, Scarpa M, Pession A, Fiori L, SIMMESN Italian Enzyme Replacement Therapy Study Group
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