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Year Number of Results
2004 1
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2011 1
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2013 3
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2015 3
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2022 3
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Page 1
A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II.
Okuyama T, Eto Y, Sakai N, Nakamura K, Yamamoto T, Yamaoka M, Ikeda T, So S, Tanizawa K, Sonoda H, Sato Y. Okuyama T, et al. Mol Ther. 2021 Feb 3;29(2):671-679. doi: 10.1016/j.ymthe.2020.09.039. Epub 2020 Sep 30. Mol Ther. 2021. PMID: 33038326 Free PMC article. Clinical Trial.
Serum HS and DS concentrations, liver and spleen volumes, and other assessments suggested the peripheral efficacy of pabinafusp alfa was comparable to that of idursulfase. Drug-related adverse events were mild or moderate in severity, transient, and manageable. ...
Serum HS and DS concentrations, liver and spleen volumes, and other assessments suggested the peripheral efficacy of pabinafusp alfa was com …
Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study.
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; HGT-HIT-094 Study Group. Muenzer J, et al. Mol Genet Metab. 2022 Sep-Oct;137(1-2):127-139. doi: 10.1016/j.ymgme.2022.07.017. Epub 2022 Aug 2. Mol Genet Metab. 2022. PMID: 36027721 Free article. Clinical Trial.
All patients continued to receive weekly intravenous idursulfase 0.5 mg/kg as standard of care. Of 49 randomized patients, 47 completed the study (two patients receiving idursulfase-IT discontinued). ...Idursulfase-IT was generally well tolerated. These data …
All patients continued to receive weekly intravenous idursulfase 0.5 mg/kg as standard of care. Of 49 randomized patients, 47 complet …
Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome).
Pano A, Barbier AJ, Bielefeld B, Whiteman DA, Amato DA. Pano A, et al. Orphanet J Rare Dis. 2015 Apr 24;10:50. doi: 10.1186/s13023-015-0265-2. Orphanet J Rare Dis. 2015. PMID: 25902842 Free PMC article. Clinical Trial.
BACKGROUND: Twenty-eight treatment-naive mucopolysaccharidosis II patients (16 months-7.5 years) received 0.5 mg/kg idursulfase weekly for one year in NCT00607386. Serum anti-idursulfase immunoglobulin G antibodies (Abs) were seen in 68% of patients. ...Liver size a …
BACKGROUND: Twenty-eight treatment-naive mucopolysaccharidosis II patients (16 months-7.5 years) received 0.5 mg/kg idursulfase weekl …
Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter syndrome).
Sohn YB, Cho SY, Park SW, Kim SJ, Ko AR, Kwon EK, Han SJ, Jin DK. Sohn YB, et al. Orphanet J Rare Dis. 2013 Mar 18;8:42. doi: 10.1186/1750-1172-8-42. Orphanet J Rare Dis. 2013. PMID: 23497636 Free PMC article. Clinical Trial.
Patients were randomized to active comparator infusions (N=11), 0.5 mg/kg idursulfase beta infusions (N=10), or 1.0 mg/kg idursulfase beta infusions (N=10). ...Both idursulfase beta infusions were generally safe and well tolerated, and elicited no serious adv …
Patients were randomized to active comparator infusions (N=11), 0.5 mg/kg idursulfase beta infusions (N=10), or 1.0 mg/kg idursulf …
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. ...Participants were also enrolled in this extension from a linked non-randomized sub-study of children younger than 3 year …
A formulation of idursulfase for intrathecal administration (idursulfase-IT) is under investigation for the treatment of neuro …
Long-term open-label phase I/II extension study of intrathecal idursulfase-IT in the treatment of neuronopathic mucopolysaccharidosis II.
Muenzer J, Vijayaraghavan S, Stein M, Kearney S, Wu Y, Alexanderian D. Muenzer J, et al. Genet Med. 2022 Jul;24(7):1437-1448. doi: 10.1016/j.gim.2022.04.002. Epub 2022 May 20. Genet Med. 2022. PMID: 35588317 Free article. Clinical Trial.
PURPOSE: Intrathecal (IT) idursulfase-IT for the treatment of cognitive impairment is being investigated in pediatric patients with neuronopathic mucopolysaccharidosis II (MPS II) in addition to intravenous idursulfase. ...RESULTS: In total, 15 participants received …
PURPOSE: Intrathecal (IT) idursulfase-IT for the treatment of cognitive impairment is being investigated in pediatric patients with n …
The relationship between anti-idursulfase antibody status and safety and efficacy outcomes in attenuated mucopolysaccharidosis II patients aged 5 years and older treated with intravenous idursulfase.
Barbier AJ, Bielefeld B, Whiteman DA, Natarajan M, Pano A, Amato DA. Barbier AJ, et al. Mol Genet Metab. 2013 Nov;110(3):303-10. doi: 10.1016/j.ymgme.2013.08.002. Epub 2013 Aug 9. Mol Genet Metab. 2013. PMID: 23988379 Free article. Clinical Trial.
In the pivotal phase II/III trial of idursulfase administered intravenously to treat mucopolysaccharidosis II, approximately half of the patients developed antibodies to idursulfase. ...A total of 63 treatment-naive patients received 0.5 mg/kg of intravenous idur …
In the pivotal phase II/III trial of idursulfase administered intravenously to treat mucopolysaccharidosis II, approximately half of …
A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4-7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy.
Giugliani R, Hwu WL, Tylki-Szymanska A, Whiteman DA, Pano A. Giugliani R, et al. Genet Med. 2014 Jun;16(6):435-41. doi: 10.1038/gim.2013.162. Epub 2013 Nov 7. Genet Med. 2014. PMID: 24202085 Free PMC article. Clinical Trial.
PURPOSE: The primary objective of this study was to determine the safety of idursulfase in Hunter syndrome patients aged 5 years or younger. METHODS: Idursulfase (0.5 mg/kg) was administered intravenously on a weekly basis (52 infusions per patient) in an open-label …
PURPOSE: The primary objective of this study was to determine the safety of idursulfase in Hunter syndrome patients aged 5 years or y …
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 …
Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome.
Muenzer J, Beck M, Eng CM, Giugliani R, Harmatz P, Martin R, Ramaswami U, Vellodi A, Wraith JE, Cleary M, Gucsavas-Calikoglu M, Puga AC, Shinawi M, Ulbrich B, Vijayaraghavan S, Wendt S, Conway AM, Rossi A, Whiteman DA, Kimura A. Muenzer J, et al. Genet Med. 2011 Feb;13(2):95-101. doi: 10.1097/GIM.0b013e3181fea459. Genet Med. 2011. PMID: 21150784 Free article. Clinical Trial.
PURPOSE: This study evaluated the safety and effectiveness of long-term enzyme replacement therapy with idursulfase (recombinant human iduronate-2-sulfatase) in patients with Hunter syndrome. METHODS: All 94 patients who completed a 53-week double-blinded study of idurs …
PURPOSE: This study evaluated the safety and effectiveness of long-term enzyme replacement therapy with idursulfase (recombinant huma …
19 results