Enzymatic replacement therapy for Hunter disease: Up to 9years experience with 17 patients-Reference-Cited by-同舟云学术

Enzymatic replacement therapy for Hunter disease: Up to 9years experience with 17 patients

Published:2015-06 Issue: Volume:3 Page:65-74
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Parini Rossella,Rigoldi Miriam,Tedesco Lucia,Boffi Lucia,Brambilla Alessandra,Bertoletti Sara,Boncimino Agata,Del Longo Alessandra,De Lorenzo Paola,Gaini Renato,Gallone Denise,Gasperini Serena,Giussani Carlo,Grimaldi Marco,Grioni Daniele,Meregalli Pamela,Messinesi Grazia,Nichelli Francesca,Romagnoli Marco,Russo Pierluigi,Sganzerla Erik,Valsecchi Grazia,Biondi Andrea

Funder

Fondazione Pierfranco and Luisa Mariani, Milano

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference44 articles.

1. Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease;Scarpa;Orphanet J. Rare Dis.,2011

2. Mucopolysaccharide diseases: a complex interplay between neuroinflammation, microglial activation and adaptive immunity;Archer;J. Inherit. Metab. Dis.,2014

3. Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome);Martin;Pediatrics,2008

4. Mucopolysaccharidosis type II;Scarpa,2007

5. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome);Muenzer;Genet. Med.,2006

Cited by 68 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and Beyond;BioDrugs;2024-08-23

2. Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice;Molecular Therapy - Methods & Clinical Development;2024-03

3. Ommaya reservoir placement using ultrasound guidance via anterior fontanelle combined with frameless electromagnetic neuronavigation in patients with mucopolysaccharidosis type 2: Case reports and review of the literature;Child's Nervous System;2024-01-02

4. Characterization of orthopedic manifestations in patients with mucopolysaccharidosis II using data from 15 years of the Hunter Outcome Survey;JIMD Reports;2023-11-27

5. Transferrin Receptor-Targeted Iduronate-2-sulfatase Penetrates the Blood-Retinal Barrier and Improves Retinopathy in Mucopolysaccharidosis II Mice;Molecular Pharmaceutics;2023-10-20