The youngest pair of siblings with Mucopolysaccharidosis type IVA to receive enzyme replacement therapy to date: A case report.
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| Title: | The youngest pair of siblings with Mucopolysaccharidosis type IVA to receive enzyme replacement therapy to date: A case report. |
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| Authors: | Frigeni, Marta1, Rodriguez‐Buritica, David F.1, Saavedra, Heather1, Gunther, Kathryn A.1, Hillman, Paul R.1, paul.hillman@uth.tmc.edu, Balaguru, Duraisamy2,3, Northrup, Hope1 |
| Source: | American Journal of Medical Genetics. Part A; Nov2021, Vol. 185 Issue 11, p3510-3516, 7p |
| Database: | Applied Science & Technology Source |
| ISSN: | 15524825 |
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| DOI: | 10.1002/ajmg.a.62469 |