Improved outcomes in infantile-onset Pompe disease through early enzyme replacement therapy enabled by newborn screening: Insights from a multisite US cohort
| Title: | Improved outcomes in infantile-onset Pompe disease through early enzyme replacement therapy enabled by newborn screening: Insights from a multisite US cohort |
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| Authors: | Desai, Ankit K. a; Rodriguez-Rassi, Eleanor a; Parikh, Suhag b; Russo, Rossana Sanchez b; Hamm, J. Austin c; Chang, Irene d; Ortiz, Damara e; McPheron, Molly f; Lydigsen, Holly g; DeArmey, Stephanie a; Young, Sarah a; Kishnani, Priya a |
| Source: | In Molecular Genetics and Metabolism February 2026 147(2) |
| Database: | ScienceDirect |