Experience with enzyme replacement therapy in children with late-onset Pompe disease diagnosed via newborn screening in the United States
Huggins, Erin, Nading, Erica, Makhijani, Neelam, Blount, Janet, Holland, Maggie, Case, Laura, Kishnani, Priya
Published in Molecular genetics and metabolism (01.02.2024)
Published in Molecular genetics and metabolism (01.02.2024)
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Journal Article
Longitudinal follow up uncovers an early emerging phenotype in children with late-onset Pompe disease diagnosed via newborn screening
Huggins, Erin, Makhijani, Neelam A., Holland, Maggie, Jones, Harrison, Blount, Janet, Jung, Seung-Hye, Case, Laura, Kishnani, Priya S.
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
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Journal Article