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A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA
The Journal of Pediatrics
◽
10.1016/j.jpeds.2015.11.079
◽
2016
◽
Vol 170
◽
pp. 278-287.e4
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Cited By ~ 45
Author(s):
Elsa G. Shapiro
◽
Igor Nestrasil
◽
Kathleen A. Delaney
◽
Kyle Rudser
◽
Victor Kovac
◽
...
Keyword(s):
Natural History
◽
Mucopolysaccharidosis Type
◽
Natural History Study
◽
Type Iiia
◽
Mucopolysaccharidosis Type Iiia
Download Full-text
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References
An observational, prospective, multicenter, natural history study of patients with mucopolysaccharidosis type IIIA
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2021.12.002
◽
2021
◽
Author(s):
Frits A. Wijburg
◽
Karen Aiach
◽
Anupam Chakrapani
◽
Julie B. Eisengart
◽
Roberto Giugliani
◽
...
Keyword(s):
Natural History
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Mucopolysaccharidosis Type
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Natural History Study
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Type Iiia
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Mucopolysaccharidosis Type Iiia
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Natural history of siblings with mucopolysaccharidosis type IIIA, Sanfilippo syndrome type A
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2016.11.127
◽
2017
◽
Vol 120
(1-2)
◽
pp. S57-S58
Author(s):
Alexandra Gossler
◽
Anja Koehn
◽
Ann Meyer
◽
Kai Kossow
◽
Kurt Ullrich
◽
...
Keyword(s):
Natural History
◽
Type A
◽
Syndrome Type
◽
Mucopolysaccharidosis Type
◽
Sanfilippo Syndrome
◽
Type Iiia
◽
History Of
◽
Mucopolysaccharidosis Type Iiia
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Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA
Case Medical Research
◽
10.31525/ct1-nct04201405
◽
2019
◽
Author(s):
Keyword(s):
Stem Cells
◽
Gene Therapy
◽
Hematopoietic Stem Cells
◽
Mucopolysaccharidosis Type
◽
Hematopoietic Stem
◽
Type Iiia
◽
Mucopolysaccharidosis Type Iiia
Download Full-text
Cardiac disease as the presenting feature of mucopolysaccharidosis type IIIA: A case report
Molecular Genetics and Metabolism Reports
◽
10.1016/j.ymgmr.2014.09.003
◽
2014
◽
Vol 1
◽
pp. 422-424
◽
Cited By ~ 5
Author(s):
Erlane Marques Ribeiro
◽
Ana Carolina Brusius-Facchin
◽
Sandra Leistner-Segal
◽
Carlos Antônio Bruno da Silva
◽
Ida Vanessa Schwartz
Keyword(s):
Case Report
◽
Cardiac Disease
◽
Mucopolysaccharidosis Type
◽
Type Iiia
◽
Mucopolysaccharidosis Type Iiia
Download Full-text
Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral-mediated gene delivery
The Journal of Gene Medicine
◽
10.1002/jgm.2816
◽
2014
◽
Vol 16
(11-12)
◽
pp. 374-387
◽
Cited By ~ 10
Author(s):
Chantelle McIntyre
◽
Ainslie L. K. Derrick-Roberts
◽
Sharon Byers
◽
Donald S. Anson
Keyword(s):
Central Nervous System
◽
Nervous System
◽
Gene Delivery
◽
Mucopolysaccharidosis Type
◽
Central Nervous System Pathology
◽
Type Iiia
◽
Mucopolysaccharidosis Type Iiia
Download Full-text
Characterization of Sulfated Oligosaccharides in Mucopolysaccharidosis Type IIIA by Electrospray Ionization Mass Spectrometry
Analytical Chemistry
◽
10.1021/ac052083d
◽
2006
◽
Vol 78
(13)
◽
pp. 4534-4542
◽
Cited By ~ 28
Author(s):
Kerryn E. Mason
◽
Peter J. Meikle
◽
John J. Hopwood
◽
Maria Fuller
Keyword(s):
Mass Spectrometry
◽
Electrospray Ionization
◽
Electrospray Ionization Mass Spectrometry
◽
Ionization Mass Spectrometry
◽
Ionization Mass
◽
Mucopolysaccharidosis Type
◽
Sulfated Oligosaccharides
◽
Type Iiia
◽
Mucopolysaccharidosis Type Iiia
Download Full-text
Neuronal-specific impairment of heparan sulfate degradation in Drosophila reveals pathogenic mechanisms for Mucopolysaccharidosis type IIIA
Experimental Neurology
◽
10.1016/j.expneurol.2018.01.020
◽
2018
◽
Vol 303
◽
pp. 38-47
◽
Cited By ~ 7
Author(s):
Dani L. Webber
◽
Amanda Choo
◽
Laura J. Hewson
◽
Paul J. Trim
◽
Marten F. Snel
◽
...
Keyword(s):
Heparan Sulfate
◽
Mucopolysaccharidosis Type
◽
Pathogenic Mechanisms
◽
Type Iiia
◽
Mucopolysaccharidosis Type Iiia
Download Full-text
Interim results of Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA)
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2019.11.129
◽
2020
◽
Vol 129
(2)
◽
pp. S56-S57
◽
Cited By ~ 1
Author(s):
Kevin M. Flanigan
◽
Nicholas J.C. Smith
◽
Maria Luz Couce
◽
Kristen V. Truxal
◽
Kim L. McBride
◽
...
Keyword(s):
Clinical Trial
◽
Gene Therapy
◽
Single Dose
◽
Phase 1
◽
Type A
◽
Syndrome Type
◽
Mucopolysaccharidosis Type
◽
Sanfilippo Syndrome
◽
Type Iiia
◽
Mucopolysaccharidosis Type Iiia
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Design and rationale of the LYS-SAF302 gene therapy study in mucopolysaccharidosis type IIIA (MPS IIIA) children
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2018.12.283
◽
2019
◽
Vol 126
(2)
◽
pp. S111-S112
Author(s):
Sophie Olivier
◽
Samantha Parker
◽
Anupam Chakrapani
◽
Ronald Crystal
◽
Lisa Emrick
◽
...
Keyword(s):
Gene Therapy
◽
Mucopolysaccharidosis Type
◽
Type Iiia
◽
Mps Iiia
◽
Mucopolysaccharidosis Type Iiia
Download Full-text
Pentosan Polysulfate Treatment of Mucopolysaccharidosis Type IIIA Mice
JIMD Reports - JIMD Reports, Volume 43
◽
10.1007/8904_2018_96
◽
2018
◽
pp. 37-52
◽
Cited By ~ 4
Author(s):
Ningning Guo
◽
Victor DeAngelis
◽
Changzhi Zhu
◽
Edward H. Schuchman
◽
Calogera M. Simonaro
Keyword(s):
Mucopolysaccharidosis Type
◽
Pentosan Polysulfate
◽
Type Iiia
◽
Mucopolysaccharidosis Type Iiia
Download Full-text
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