Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial-Reference-Cited by-同舟云学术

Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial

Published:2019-02 Issue:2 Volume:126 Page:121-130
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Wijburg Frits A.,Whitley Chester B.,Muenzer Joseph,Gasperini Serena^ORCID,del Toro Mireia^ORCID,Muschol Nicole,Cleary Maureen,Sevin Caroline,Shapiro Elsa^ORCID,Bhargava Parul,Kerr Douglas,Alexanderian David

Publisher

Elsevier BV

Subject

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

Reference13 articles.

1. Mental retardation associated with acid mucopolysacchariduria (heparitin sulfate type);Sanfilippo;J. Pediatr.,1963

2. Cognitive development in patients with Mucopolysaccharidosis type III (Sanfilippo syndrome);Valstar;Orphanet J. Rare Dis.,2011

3. Sanfilippo syndrome: causes, consequences, and treatments;Fedele;Appl. Clin. Genet.,2015

4. Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations;Valstar;Ann. Neurol.,2010

5. Prospective Natural history Study of Mucopolysaccharidosis Type IIIA;Shapiro;J. Pediatr.,2016

Cited by 38 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. Evaluation of neuroretina following i.v. or intra‐CSF AAV9 gene replacement in mice with MPS IIIA, a childhood dementia;CNS Neuroscience & Therapeutics;2024-08

2. Current Concepts in the Management of Sanfilippo Syndrome (MPS III): A Narrative Review;Cureus;2024-04-11

3. Drosophila melanogaster models of MPS IIIC (Hgsnat‐deficiency) highlight the role of glia in disease presentation;Journal of Inherited Metabolic Disease;2024-01-18

4. Repetitive, non-invasive imaging of neurodegeneration, and prevention of it with gene replacement, in mice with Sanfilippo syndrome.;Experimental Neurology;2024-01

5. Clinical, biochemical, and molecular characterization of mucopolysaccharidosis type III in 34 Egyptian patients;American Journal of Medical Genetics Part A;2023-07-10