Enzyme replacement therapy in infants and very young children with Gaucher disease using velaglucerase alfa: a single-center experience.
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| Title: | Enzyme replacement therapy in infants and very young children with Gaucher disease using velaglucerase alfa: a single-center experience. |
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| Authors: | Goker-Alpan O; Lysosomal & Rare Disorders Research & Treatment Center, Fairfax, VA, United States., Ivanova MM; Lysosomal & Rare Disorders Research & Treatment Center, Fairfax, VA, United States., Pathak R; Takeda Pharmaceuticals USA, Inc., Lexington, MA, United States., Wright E; Takeda Pharmaceuticals USA, Inc., Lexington, MA, United States. |
| Source: | Frontiers in pediatrics [Front Pediatr] 2025 Oct 17; Vol. 13, pp. 1613599. Date of Electronic Publication: 2025 Oct 17 (Print Publication: 2025). |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Frontiers Media SA Country of Publication: Switzerland NLM ID: 101615492 Publication Model: eCollection Cited Medium: Print ISSN: 2296-2360 (Print) Linking ISSN: 22962360 NLM ISO Abbreviation: Front Pediatr Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 2296-2360 |
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| DOI: | 10.3389/fped.2025.1613599 |
