Long-term open-label phase I/II extension study of intrathecal idursulfase-IT in the treatment of neuronopathic mucopolysaccharidosis II
| Autor: | Muenzer, Joseph, Vijayaraghavan, Suresh, Stein, Margot, Kearney, Shauna, Wu, Yuna, Alexanderian, David |
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| Zdroj: | In Genetics in Medicine July 2022 24(7):1437-1448 |
| Databáze: | ScienceDirect |
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