Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment.
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| Title: | Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment. |
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| Authors: | Yee, Karen S.1,2 (AUTHOR), Alexanderian, David3,4 (AUTHOR), Martin, Susan5 (AUTHOR), Olayinka-Amao, Bimpe6 (AUTHOR), Whiteman, David A. H.3 (AUTHOR) dahw.md@gmail.com |
| Source: | Orphanet Journal of Rare Diseases. 4/12/2024, Vol. 19 Issue 1, p1-11. 11p. |
| Database: | Academic Search Ultimate |
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| ISSN: | 17501172 |
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| DOI: | 10.1186/s13023-024-03147-4 |
