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Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C
CASE: A child with Niemann-Pick disease type C was started on miglustat therapy at the age of 2 years. Intrathecal administration of hydroxypropyl-β-cyclodextrin was added 5 months later. The initial dose of 175 mg was gradually increased over the first 6 months to reach 325 mg. The drug was adminis...
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| Publicat a: | Eur J Hosp Pharm |
|---|---|
| Autors principals: | , , , , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
BMJ Publishing Group
2017
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6451626/ https://ncbi.nlm.nih.gov/pubmed/31156935 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/ejhpharm-2016-001067 |
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