Optimizing clinical outcomes: The journey of twins with CRIM-negative infantile-onset Pompe disease on high-dose enzyme replacement therapy and immunomodulation
| Title: | Optimizing clinical outcomes: The journey of twins with CRIM-negative infantile-onset Pompe disease on high-dose enzyme replacement therapy and immunomodulation |
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| Authors: | Fares, Angie H., Desai, Ankit K., Case, Laura E., Sharon, Cassie, Klinepeter, Amy, Kirby, Amelia, Lisi, Matthew T., Koch, Rebecca L., Kishnani, Priya S. |
| Source: | In Molecular Genetics and Metabolism Reports December 2024 41 |
| Database: | ScienceDirect |
| ISSN: | 22144269 |
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| DOI: | 10.1016/j.ymgmr.2024.101141 |
| Published in: | Molecular Genetics and Metabolism Reports |
| Language: | English |