Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen-Reference-Cited by-同舟云学术

Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen

Published:2010-02 Issue:2 Volume:99 Page:132-141
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Auclair Dyane,Finnie John,White Joleen,Nielsen Timothy,Fuller Maria,Kakkis Emil,Cheng Alphonsus,O’Neill Charles A.,Hopwood John J.

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference55 articles.

1. The mucopolysaccharidoses;Neufeld,1989

2. Bone marrow transplantation in the Maroteaux–Lamy syndrome (mucopolysaccharidosis yype VI) biochemical and clinical status 24months after transplantation;Krivit;N. Engl. J. Med.,1984

3. Stem cell bone marrow transplantation in patients with metabolic storage diseases;Krivit;Adv. Pediatr.,2002

4. Bone marrow transplantation for Maroteaux–Lamy syndrome (MPS-VI): long term follow up;Herskhovitz;J. Inherit. Metab. Dis.,1999

5. MPS VI Study Group Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase;Harmatz;Mol. Genet. Metab.,2008

Cited by 35 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. Quantification of Glycosaminoglycans in Urine by Isotope‐Dilution Liquid Chromatography‐Electrospray Ionization Tandem Mass Spectrometry;Current Protocols;2023-03

2. Transduction characteristics of alternative adeno-associated virus serotypes in the cat brain by intracisternal delivery;Molecular Therapy - Methods & Clinical Development;2022-09

3. Mucopolysaccharidoses;Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics;2021

4. Novel therapies for mucopolysaccharidosis type III;Journal of Inherited Metabolic Disease;2020-09-28

5. New treatment method for mucopolysaccharidosis type VI by liver transplantation;Pediatrics International;2018-12-13