Search research articles
Contact Us
Filters
Showing results (61-70 of 93) with videos related to
Page
of 10
Sort By:
Journal of Inherited Metabolic Disease
|
June 11, 2026
Validating a Human Cell Model of Null Galactosylceramidase (GALC) Enzyme Activity That Recapitulates Krabbe Disease
Rodrigo T Starosta, Nazia Khatoon, Marie S Roberts, et al.
JIMD Reports
|
February 23, 2013
Mannose 6-phosphate conjugation is not sufficient to allow induction of immune tolerance to phenylalanine ammonia-lyase in dogs
Moin Vera, Thomas Lester, Bin Zhao, et al.
American Journal of Medical Genetics. Part A
|
February 22, 2020
Phenotypic expansion of KMT2D-related disorder: Beyond Kabuki syndrome
Dustin Baldridge, Rebecca C Spillmann, Daniel J Wegner, et al.
Molecular Pharmaceutics
|
December 15, 2020
Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-<i>N</i>-Acetylglucosamine-6-Sulfatase in Neonatal Mice
Feng Wang, Derek R Moen, Chelsee Sauni, et al.
Molecular Genetics and Metabolism
|
May 12, 2024
D-mannose as a new therapy for fucokinase deficiency-related congenital disorder of glycosylation (FCSK-CDG)
Rodrigo Tzovenos Starosta, Angela J Lee, Elizabeth R Toolan, et al.
Molecular Therapy. Methods & Clinical Development
|
November 22, 2017
A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I
Steven Q Le, Shih-Hsin Kan, Don Clarke, et al.
Molecular Genetics and Metabolism
|
June 22, 2014
Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I
Raymond Y Wang, Afshin Aminian, Michael F McEntee, et al.
Environmental Pollution (Barking, Essex : 1987)
|
October 24, 2006
Reducing fluxes of faecal indicator compliance parameters to bathing waters from diffuse agricultural sources: the Brighouse Bay study, Scotland
D Kay, M Aitken, J Crowther, et al.
Molecular Genetics and Metabolism Reports
|
June 11, 2021
A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-β-cyclodextrin for acute liver disease in infants with Niemann-Pick C1
Margaret Reynolds, Laura A Linneman, Sofia Luna, et al.
Ethnicity & Disease
|
September 12, 2018
Community Partnership in Precision Medicine: Themes from a Community Engagement Conference
Loretta Jones, Kenneth Wells, Henry J Lin, et al.
Page
of 10
Search research articles
Search
Showing results (61-70 of 93) with videos related to
Sort By:
Page
of 10
Journal of Inherited Metabolic Disease
|
June 11, 2026
Validating a Human Cell Model of Null Galactosylceramidase (GALC) Enzyme Activity That Recapitulates Krabbe Disease
Rodrigo T Starosta, Nazia Khatoon, Marie S Roberts, et al.
JIMD Reports
|
February 23, 2013
Mannose 6-phosphate conjugation is not sufficient to allow induction of immune tolerance to phenylalanine ammonia-lyase in dogs
Moin Vera, Thomas Lester, Bin Zhao, et al.
American Journal of Medical Genetics. Part A
|
February 22, 2020
Phenotypic expansion of KMT2D-related disorder: Beyond Kabuki syndrome
Dustin Baldridge, Rebecca C Spillmann, Daniel J Wegner, et al.
Molecular Pharmaceutics
|
December 15, 2020
Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-<i>N</i>-Acetylglucosamine-6-Sulfatase in Neonatal Mice
Feng Wang, Derek R Moen, Chelsee Sauni, et al.
Molecular Genetics and Metabolism
|
May 12, 2024
D-mannose as a new therapy for fucokinase deficiency-related congenital disorder of glycosylation (FCSK-CDG)
Rodrigo Tzovenos Starosta, Angela J Lee, Elizabeth R Toolan, et al.
Molecular Therapy. Methods & Clinical Development
|
November 22, 2017
A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I
Steven Q Le, Shih-Hsin Kan, Don Clarke, et al.
Molecular Genetics and Metabolism
|
June 22, 2014
Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I
Raymond Y Wang, Afshin Aminian, Michael F McEntee, et al.
Environmental Pollution (Barking, Essex : 1987)
|
October 24, 2006
Reducing fluxes of faecal indicator compliance parameters to bathing waters from diffuse agricultural sources: the Brighouse Bay study, Scotland
D Kay, M Aitken, J Crowther, et al.
Molecular Genetics and Metabolism Reports
|
June 11, 2021
A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-β-cyclodextrin for acute liver disease in infants with Niemann-Pick C1
Margaret Reynolds, Laura A Linneman, Sofia Luna, et al.
Ethnicity & Disease
|
September 12, 2018
Community Partnership in Precision Medicine: Themes from a Community Engagement Conference
Loretta Jones, Kenneth Wells, Henry J Lin, et al.
Page
of 10