.Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AB05_Laronidase.Laronidase .Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AB05_Laronidase.Laronidase| name: | Laronidase |
| ATC code: | A16AB05 | route: | intravenous |
| n-compartments | 2 |
Laronidase is a recombinant human alpha-L-iduronidase, an enzyme replacement therapy approved for the treatment of mucopolysaccharidosis I (MPS I, Hurler, Hurler-Scheie, and Scheie syndromes), a rare lysosomal storage disorder. Laronidase helps reduce non-neurological symptoms related to glycosaminoglycan accumulation in affected individuals. It is currently approved and used for MPS I treatment.
Pharmacokinetic parameters reported in pediatric and adult patients with MPS I (non-neurological). Study population: 8 children (aged 5–12 years), 8 adults (aged 15–43 years).
Schuh, RS, et al., & Ferreira Teixeira, H (2024). Laronidase-loaded liposomes reach the brain and other hard-to-treat organs after noninvasive nasal administration. International journal of pharmaceutics 660 124355–None. DOI:10.1016/j.ijpharm.2024.124355 PUBMED:https://pubmed.ncbi.nlm.nih.gov/38897489