دورية أكاديمية
Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II
العنوان: | Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II |
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المؤلفون: | Yee, Karen S.Aff1, IDs1302302403034y_cor1, Lewis, Sandy, Evans, Emily, Romano, Carla, Alexanderian, David |
المصدر: | Orphanet Journal of Rare Diseases. 19(1) |
قاعدة البيانات: | Springer Nature Journals |
تدمد: | 17501172 |
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DOI: | 10.1186/s13023-024-03034-y |