Real-life Impacts of Olipudase Alfa: the Experience of Patients and Families Taking an Enzyme Replacement Therapy for Acid Sphingomyelinase Deficiency
ORPHANET JOURNAL OF RARE DISEASES（2024）
关键词
Acid sphingomyelinase deficiency,Olipudase alfa,Enzyme replacement therapy,Niemann Pick diseases,Type A,Type A/B,Type B,Visceral symptoms,Neurologic symptoms
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