A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II.
Muenzer J, Hendriksz CJ, Fan Z, Vijayaraghavan S, Perry V, Santra S, Solanki GA, Mascelli MA, Pan L, Wang N, Sciarappa K, Barbier AJ.
Muenzer J, et al.
Genet Med. 2016 Jan;18(1):73-81. doi: 10.1038/gim.2015.36. Epub 2015 Apr 2.
Genet Med. 2016.
PMID: 25834948
Free article.
Clinical Trial.
We examined the safety of idursulfase formulated for intrathecal administration (idursulfase-IT) via intrathecal drug delivery device (IDDD). ...METHODS: Sixteen cognitively impaired males with mucopolysaccharidosis II who were previously treated with weekly i.v. …
We examined the safety of idursulfase formulated for intrathecal administration (idursulfase-IT) via intrathecal drug delivery …