دورية أكاديمية
Real-life impacts of olipudase alfa: The experience of patients and families taking an enzyme replacement therapy for acid sphingomyelinase deficiency
العنوان: | Real-life impacts of olipudase alfa: The experience of patients and families taking an enzyme replacement therapy for acid sphingomyelinase deficiency |
---|---|
المؤلفون: | Raebel, Eva M., Wiseman, Samantha, Donnelly, ConanAff2, IDs13023024030204_cor3, Mathieson, Toni, Pountney, Jackson, Crowe, Joslyn, Hopkin, Justin |
المصدر: | Orphanet Journal of Rare Diseases. 19(1) |
قاعدة البيانات: | Springer Nature Journals |
تدمد: | 17501172 |
---|---|
DOI: | 10.1186/s13023-024-03020-4 |