Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB-Reference-Cited by-同舟云学术

Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB

Published:2022-10 Issue: Volume:249 Page:50-58.e2
ISSN:0022-3476
Container-title:The Journal of Pediatrics
language:en
Short-container-title:The Journal of Pediatrics

Author:

Okur Ilyas,Ezgu Fatih,Giugliani Roberto,Muschol Nicole,Koehn Anja,Amartino Hernan,Harmatz Paul,de Castro Lopez Maria J.,Couce Maria Luz,Lin Shuan-Pei,Batzios Spyros,Cleary Maureen,Solano Martha,Peters Heidi,Lee Joy,Nestrasil Igor,Shaywitz Adam J.,Maricich Stephen M.,Kuca Bernice,Kovalchin Joseph,Zanelli Eric

Publisher

Elsevier BV

Subject

Pediatrics, Perinatology and Child Health

Reference40 articles.

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2. Epidemiology of mucopolysaccharidoses (MPS) in United States: challenges and opportunities;Puckett;Orphanet J Rare Dis,2021

3. Mucopolysaccharidosis III in Taiwan: natural history, clinical and molecular characteristics of 28 patients diagnosed during a 21-year period;Lin;Am J Med Genet A,2018

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

5. Mortality in patients with Sanfilippo syndrome;Lavery;Orphanet J Rare Dis,2017

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1. Health service utilization, economic burden and quality of life of patients with mucopolysaccharidosis in China;Orphanet Journal of Rare Diseases;2024-09-06

2. Community consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic Mucopolysaccharidoses;Molecular Genetics and Metabolism;2024-08

3. Disease pathology signatures in a mouse model of Mucopolysaccharidosis type IIIB;Scientific Reports;2023-10-04

4. Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape;Neural Regeneration Research;2023-05-31

5. A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B;Journal of Clinical Investigation;2023-01-17