Observational Prospective Natural History of Patients with Sanfilippo Syndrome Type B-Reference-Cited by-同舟云学术

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Observational Prospective Natural History of Patients with Sanfilippo Syndrome Type B

Published:2018-06 Issue: Volume:197 Page:198-206.e2
ISSN:0022-3476
Container-title:The Journal of Pediatrics
language:en
Short-container-title:The Journal of Pediatrics

Author:

Whitley Chester B.,Cleary Maureen,Eugen Mengel Karl,Harmatz Paul,Shapiro Elsa,Nestrasil Igor,Haslett Patrick,Whiteman David,Alexanderian David

Funder

Shire

Lysosomal Disease Network

CTSI

Alexion

Armagen

BioMarin Pharmaceutical

Genzyme Sanofi

Inventiva

Sangamo

Ultragenyx

Publisher

Elsevier BV

Subject

Pediatrics, Perinatology, and Child Health

Reference38 articles.

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

2. Prevalence of lysosomal storage disorders;Meikle;JAMA,1999

3. Cumulative incidence rates of the mucopolysaccharidoses in Germany;Baehner;J Inherit Metab Dis,2005

4. The frequency of lysosomal storage diseases in The Netherlands;Poorthuis;Hum Genet,1999

5. Incidence of the mucopolysaccharidoses in Taiwan, 1984-2004;Lin;Am J Med Genet A,2009

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1. Anakinra in Sanfilippo syndrome: a phase 1/2 trial;Nature Medicine;2024-06-21

2. Psychobehavioral factors and family functioning in mucopolysaccharidosis: preliminary studies;Frontiers in Public Health;2024-01-24

3. Disease progression in Sanfilippo type B: Case series of Brazilian patients;Genetics and Molecular Biology;2024

4. Severe central nervous system demyelination in Sanfilippo disease;Frontiers in Molecular Neuroscience;2023-12-13

5. Adaptive skills in children with mucopolysaccharidoses;L.O. Badalyan Neurological Journal;2023-04-20

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