Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II.
Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D; SHP609-302 study group.
Muenzer J, et al.
Mol Genet Metab. 2022 Sep-Oct;137(1-2):92-103. doi: 10.1016/j.ymgme.2022.07.016. Epub 2022 Aug 2.
Mol Genet Metab. 2022.
PMID: 35961250
Free article.
Clinical Trial.
A formulation of idursulfase for intrathecal administration (idursulfase-IT) is under investigation for the treatment of neuronopathic MPS II. ...Overall, 25.0% of patients had at least one adverse event (AE) related to idursulfase-IT; most trea …
A formulation of idursulfase for intrathecal administration (idursulfase-IT) is under investigation for the treatment of neuro …