Long-term outcomes of imiglucerase treatment in children with Gaucher disease type 1 or type 3 starting therapy before the age of 2 years
| Title: | Long-term outcomes of imiglucerase treatment in children with Gaucher disease type 1 or type 3 starting therapy before the age of 2 years |
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| Authors: | Mistry, Pramod K. a, Carwile, Jenny L. b, Burrow, Andrew c, Ganesh, Jaya d, Camelo, José Simon, Jr e, Hennermann, Julia B. f, Batista, Julie L. b, Oliveira-dos-Santos, Antonio g, Perichon, Maria G. b, Tantawy, Azza h |
| Source: | In Molecular Genetics and Metabolism February 2025 144(2) |
| Database: | ScienceDirect |
| ISSN: | 10967192 |
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| DOI: | 10.1016/j.ymgme.2024.108858 |
| Published in: | Molecular Genetics and Metabolism |
| Language: | English |