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Natural history of Sanfilippo syndrome type A
Journal of Inherited Metabolic Disease
◽
10.1007/s10545-013-9661-8
◽
2013
◽
Vol 37
(3)
◽
pp. 431-437
◽
Cited By ~ 45
Author(s):
Dakota Buhrman
◽
Kavita Thakkar
◽
Michele Poe
◽
Maria L. Escolar
Keyword(s):
Natural History
◽
Type A
◽
Syndrome Type
◽
Sanfilippo Syndrome
◽
History Of
Download Full-text
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Natural history of siblings with mucopolysaccharidosis type IIIA, Sanfilippo syndrome type A
Molecular Genetics and Metabolism
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10.1016/j.ymgme.2016.11.127
◽
2017
◽
Vol 120
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◽
pp. S57-S58
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Alexandra Gossler
◽
Anja Koehn
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...
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◽
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◽
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◽
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◽
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The natural history of Sanfilippo syndrome type A
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2012.11.243
◽
2013
◽
Vol 108
(2)
◽
pp. S90
Author(s):
Kavita Thakkar
◽
Dakota Buhrman
◽
Michele Poe
◽
Maria L. Escolar
Keyword(s):
Natural History
◽
Type A
◽
Syndrome Type
◽
Sanfilippo Syndrome
◽
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Design and rational of the natural history study in preparation for the pivotal clinical trial for the treatment of Sanfilippo syndrome type A
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2016.11.036
◽
2017
◽
Vol 120
(1-2)
◽
pp. S25-S26
Author(s):
Soraya Bekkali
◽
Samantha Parker
◽
Anupam Chakrapani
◽
Roberto Giugliani
◽
Benedicte Heron
◽
...
Keyword(s):
Clinical Trial
◽
Natural History
◽
Type A
◽
Syndrome Type
◽
Sanfilippo Syndrome
◽
Natural History Study
◽
Pivotal Clinical Trial
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Natural history of Sanfilippo syndrome type B in young patients: Ongoing results from two large, prospective studies
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2020.12.091
◽
2021
◽
Vol 132
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◽
pp. S44
Author(s):
Roberto Giugliani
◽
Ilyas Okur
◽
Fatih Ezgu
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Nicole Muschol
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Keyword(s):
Natural History
◽
Prospective Studies
◽
Young Patients
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Syndrome Type
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Type B
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Sanfilippo Syndrome
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Observational Prospective Natural History of Patients with Sanfilippo Syndrome Type B
The Journal of Pediatrics
◽
10.1016/j.jpeds.2018.01.044
◽
2018
◽
Vol 197
◽
pp. 198-206.e2
◽
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Author(s):
Chester B. Whitley
◽
Maureen Cleary
◽
Karl Eugen Mengel
◽
Paul Harmatz
◽
Elsa Shapiro
◽
...
Keyword(s):
Natural History
◽
Syndrome Type
◽
Type B
◽
Sanfilippo Syndrome
◽
History Of
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Natural history of Morquio syndrome type A patient with tracheal obstruction from birth to death
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2017.12.382
◽
2018
◽
Vol 123
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◽
pp. S139
Author(s):
Shunji Tomatsu
◽
Caitlin Doherty
◽
Lauren W. Averill
◽
Mary Theroux
◽
Robert W. Mason
Keyword(s):
Natural History
◽
Type A
◽
Syndrome Type
◽
Tracheal Obstruction
◽
History Of
◽
Morquio Syndrome
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Natural History of the ECG Abnormalities in FAP Portuguese Type: A Follow-Up Study with ECG, Holter Monitoring and Intracardiac Electrophysiological Study
Amyloid and Amyloidosis
◽
10.1007/978-1-4757-0298-9_76
◽
1988
◽
pp. 453-457
◽
Cited By ~ 1
Author(s):
Luiz Maciel
◽
D. Cunha
◽
A. Oliveira
◽
J. Campos
◽
Falcão de Freitas
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◽
Electrophysiological Study
◽
Type A
◽
Holter Monitoring
◽
Follow Up Study
◽
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◽
Ecg Abnormalities
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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
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10.1016/j.ymgme.2019.11.129
◽
2020
◽
Vol 129
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◽
pp. S56-S57
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Nicholas J.C. Smith
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◽
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Keyword(s):
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◽
Gene Therapy
◽
Single Dose
◽
Phase 1
◽
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◽
Syndrome Type
◽
Mucopolysaccharidosis Type
◽
Sanfilippo Syndrome
◽
Type Iiia
◽
Mucopolysaccharidosis Type Iiia
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Cardiac Ultrasound Findings in Sanfilippo Syndrome Type A
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2011.11.074
◽
2012
◽
Vol 105
(2)
◽
pp. S34
Author(s):
Brandon Hays
◽
Elizabeth Braunlin
◽
James Berry
◽
Brenda Diethelm-Okita
◽
Chester Whitley
Keyword(s):
Type A
◽
Cardiac Ultrasound
◽
Syndrome Type
◽
Sanfilippo Syndrome
◽
Ultrasound Findings
Download Full-text
Sanfilippo syndrome type A (MPS IIIA) with attenuated genotype manifesting in infancy with dysostosis multiplex
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2019.11.371
◽
2020
◽
Vol 129
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◽
pp. S141
Author(s):
Stephanie Sacharow
Keyword(s):
Type A
◽
Syndrome Type
◽
Sanfilippo Syndrome
◽
Dysostosis Multiplex
◽
Mps Iiia
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