RGX-111 GENE THERAPY FOR THE TREATMENT OF SEVERE MUCOPOLYSACCHARIDOSIS TYPE I (MPS I): INTERIM ANALYSIS OF DATA FROM THE FIRST IN HUMAN STUDY
| Autor: | Gilmor, Michelle, Wang, Raymond, Ficicioglu, Can, Giugliani, Roberto, Burke, Jenna, Fiscella, Michele, Yang, Lin, Phillips, Dawn, Falabella, Paulo |
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| Zdroj: | In Molecular Genetics and Metabolism March 2023 138(3) |
| Databáze: | ScienceDirect |
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