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Molecular Genetics and Metabolism
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December 12, 2018
Comprehensive behavioral and biochemical outcomes of novel murine models of GM1-gangliosidosis and Morquio syndrome type B
Michael J Przybilla, Li Ou, Alexandru-Flaviu Tăbăran, et al.
Molecular Genetics and Metabolism
|
February 15, 2022
Contribution of the innate and adaptive immune systems to aortic dilation in murine mucopolysaccharidosis type I
Elizabeth Braunlin, Juan E Abrahante, Ron McElmurry, et al.
Journal of Pediatric Rehabilitation Medicine
|
August 7, 2014
Biomarkers of bone remodeling in children with mucopolysaccharidosis types I, II, and VI
David A Stevenson, Kyle Rudser, Alicia Kunin-Batson, et al.
Scientific Reports
|
August 5, 2023
Mapping brain networks in MPS I mice and their restoration following gene therapy
Wei Zhu, Li Ou, Lin Zhang, et al.
Molecular Genetics and Metabolism Reports
|
April 27, 2016
A longitudinal study of emotional adjustment, quality of life and adaptive function in attenuated MPS II
Elsa G Shapiro, Kyle Rudser, Alia Ahmed, et al.
Journal of Inherited Metabolic Disease
|
January 6, 2025
Diffusion tensor imaging with free-water correction reveals distinctions between severe and attenuated subtypes in Mucopolysaccharidosis type I
Alena Svatkova, Ofer Pasternak, Julie B Eisengart, et al.
Orphanet Journal of Rare Diseases
|
October 28, 2022
Sanfilippo syndrome: consensus guidelines for clinical care
Nicole Muschol, Roberto Giugliani, Simon A Jones, et al.
Gene Therapy
|
January 4, 2020
A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases
Li Ou, Michael J Przybilla, Alexandru-Flaviu Tăbăran, et al.
Molecular Genetics and Metabolism
|
March 16, 2015
Quantifying behaviors of children with Sanfilippo syndrome: the Sanfilippo Behavior Rating Scale
Elsa G Shapiro, Igor Nestrasil, Alia Ahmed, et al.
Molecular Genetics and Metabolism
|
December 12, 2018
Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial
Frits A Wijburg, Chester B Whitley, Joseph Muenzer, et al.
Page
of 12
Search research articles
Search
Showing results (71-80 of 116) with videos related to
Sort By:
Page
of 12
Molecular Genetics and Metabolism
|
December 12, 2018
Comprehensive behavioral and biochemical outcomes of novel murine models of GM1-gangliosidosis and Morquio syndrome type B
Michael J Przybilla, Li Ou, Alexandru-Flaviu Tăbăran, et al.
Molecular Genetics and Metabolism
|
February 15, 2022
Contribution of the innate and adaptive immune systems to aortic dilation in murine mucopolysaccharidosis type I
Elizabeth Braunlin, Juan E Abrahante, Ron McElmurry, et al.
Journal of Pediatric Rehabilitation Medicine
|
August 7, 2014
Biomarkers of bone remodeling in children with mucopolysaccharidosis types I, II, and VI
David A Stevenson, Kyle Rudser, Alicia Kunin-Batson, et al.
Scientific Reports
|
August 5, 2023
Mapping brain networks in MPS I mice and their restoration following gene therapy
Wei Zhu, Li Ou, Lin Zhang, et al.
Molecular Genetics and Metabolism Reports
|
April 27, 2016
A longitudinal study of emotional adjustment, quality of life and adaptive function in attenuated MPS II
Elsa G Shapiro, Kyle Rudser, Alia Ahmed, et al.
Journal of Inherited Metabolic Disease
|
January 6, 2025
Diffusion tensor imaging with free-water correction reveals distinctions between severe and attenuated subtypes in Mucopolysaccharidosis type I
Alena Svatkova, Ofer Pasternak, Julie B Eisengart, et al.
Orphanet Journal of Rare Diseases
|
October 28, 2022
Sanfilippo syndrome: consensus guidelines for clinical care
Nicole Muschol, Roberto Giugliani, Simon A Jones, et al.
Gene Therapy
|
January 4, 2020
A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases
Li Ou, Michael J Przybilla, Alexandru-Flaviu Tăbăran, et al.
Molecular Genetics and Metabolism
|
March 16, 2015
Quantifying behaviors of children with Sanfilippo syndrome: the Sanfilippo Behavior Rating Scale
Elsa G Shapiro, Igor Nestrasil, Alia Ahmed, et al.
Molecular Genetics and Metabolism
|
December 12, 2018
Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial
Frits A Wijburg, Chester B Whitley, Joseph Muenzer, et al.
Page
of 12