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Intracerebral gene therapy for Sanfilippo syndrome type A
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2012.11.281
◽
2013
◽
Vol 108
(2)
◽
pp. S102
Author(s):
Amina Zinai
Keyword(s):
Gene Therapy
◽
Type A
◽
Syndrome Type
◽
Sanfilippo Syndrome
Download Full-text
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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)
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Keyword(s):
Clinical Trial
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Gene Therapy
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Single Dose
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Phase 1
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Type A
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Mucopolysaccharidosis Type
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Cardiac Ultrasound Findings in Sanfilippo Syndrome Type A
Molecular Genetics and Metabolism
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10.1016/j.ymgme.2011.11.074
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◽
pp. S34
Author(s):
Brandon Hays
◽
Elizabeth Braunlin
◽
James Berry
◽
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◽
Chester Whitley
Keyword(s):
Type A
◽
Cardiac Ultrasound
◽
Syndrome Type
◽
Sanfilippo Syndrome
◽
Ultrasound Findings
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Sanfilippo syndrome type A (MPS IIIA) with attenuated genotype manifesting in infancy with dysostosis multiplex
Molecular Genetics and Metabolism
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10.1016/j.ymgme.2019.11.371
◽
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Author(s):
Stephanie Sacharow
Keyword(s):
Type A
◽
Syndrome Type
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Sanfilippo Syndrome
◽
Dysostosis Multiplex
◽
Mps Iiia
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Genotype, Disease Onset, And Neurocognitive Phenotype Of Sanfilippo Syndrome Type A
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2011.11.152
◽
2012
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Vol 105
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pp. S58
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Cited By ~ 1
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Chester B. Whitley
◽
Renee Cooksley
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Brenda Diethelm-Okita
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Keyword(s):
Disease Onset
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Type A
◽
Syndrome Type
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Sanfilippo Syndrome
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Mutation and haplotype analyses in 26 Spanish Sanfilippo syndrome type A patients: Possible single origin for 1091delC mutation
American Journal of Medical Genetics
◽
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pp. 223-228
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◽
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◽
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Maria Josep Coll
◽
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Type A
◽
Syndrome Type
◽
Sanfilippo Syndrome
◽
Single Origin
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The impact on children and parents of participation in clinical research trials for Morquio syndrome type A and Sanfilippo syndrome type A
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2013.12.059
◽
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◽
Vol 111
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◽
pp. S32-S33
Author(s):
Tara Clancy
◽
Maria Farag
◽
Deborah Holliday
◽
Catherine Breen
◽
Simon Jones
Keyword(s):
Clinical Research
◽
Type A
◽
Syndrome Type
◽
Sanfilippo Syndrome
◽
Children And Parents
◽
Morquio Syndrome
◽
Clinical Research Trials
◽
The Impact
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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
◽
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◽
Benedicte Heron
◽
...
Keyword(s):
Clinical Trial
◽
Natural History
◽
Type A
◽
Syndrome Type
◽
Sanfilippo Syndrome
◽
Natural History Study
◽
Pivotal Clinical Trial
Download Full-text
Development of a stem cell gene therapy for Sanfilippo syndrome type B
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2016.11.068
◽
2017
◽
Vol 120
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◽
pp. S37
◽
Cited By ~ 1
Author(s):
Don L Clarke
◽
Valentina Sanguez
◽
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Keyword(s):
Gene Therapy
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Stem Cell
◽
Syndrome Type
◽
Type B
◽
Sanfilippo Syndrome
◽
Cell Gene
◽
Stem Cell Gene
◽
Stem Cell Gene Therapy
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Scoring Evaluation of the Natural Course of Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
PEDIATRICS
◽
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◽
2007
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◽
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Author(s):
A. Meyer
◽
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◽
A. Gal
◽
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Type A
◽
Natural Course
◽
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◽
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◽
Sanfilippo Syndrome
◽
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◽
Mucopolysaccharidosis Type Iiia
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873. Synergistic and Antagonistic Effects of Neonatal Intracranial AAV Gene Therapy and Bone Marrow Transplantation in Sanfilippo Syndrome Type B Mice
Molecular Therapy
◽
10.1016/s1525-0016(16)40276-5
◽
2008
◽
Vol 16
◽
pp. S325-S326
Keyword(s):
Gene Therapy
◽
Bone Marrow
◽
Bone Marrow Transplantation
◽
Marrow Transplantation
◽
Syndrome Type
◽
Type B
◽
Sanfilippo Syndrome
◽
Antagonistic Effects
◽
B Mice
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