Long-term safety and clinical outcomes of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A-Reference-Cited by-同舟云学术

Long-term safety and clinical outcomes of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A

Published:2021-12 Issue:4 Volume:134 Page:317-322
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Wijburg Frits A.,Heap Fiona,Rust Stewart,de Ruijter Jessica,Tump Evelien,Marchal Jan Pieter,Nestrasil Igor,Shapiro Elsa,Jones Simon A.,Alexanderian David

Publisher

Elsevier BV

Subject

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

Reference13 articles.

1. Newborn screening and diagnosis of mucopolysaccharidoses;Tomatsu;Mol. Genet. Metab.,2013

2. Epidemiology of Sanfilippo syndrome: results of a systematic literature review;Zelei;Orphanet J. Rare Dis.,2018

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

4. A prospective natural history study of mucopolysaccharidosis type IIIA;Shapiro;J. Pediatr.,2016

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

Cited by 5 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. The Role of the Gut Microbiota in Sanfilippo Syndrome’s Physiopathology: An Approach in Two Affected Siblings;International Journal of Molecular Sciences;2024-08-14

2. Molecular Mechanisms in Pathophysiology of Mucopolysaccharidosis and Prospects for Innovative Therapy;International Journal of Molecular Sciences;2024-01-17

3. Patients’ view on gene therapy development for lysosomal storage disorders: a qualitative study;Orphanet Journal of Rare Diseases;2022-10-21

4. Sanfilippo Syndrome: Optimizing Care with a Multidisciplinary Approach;Journal of Multidisciplinary Healthcare;2022-09

5. Advancing the Research and Development of Enzyme Replacement Therapies for Lysosomal Storage Diseases;GEN Biotechnology;2022-04-01