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Pediatric Research
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August 22, 2019
Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation
Lynda E Polgreen, Troy C Lund, Elizabeth Braunlin, et al.
Molecular Genetics and Metabolism
|
April 11, 2025
Safety and efficacy of migalastat in adolescent patients with Fabry disease: Results from ASPIRE, a phase 3b, open-label, single-arm, 12-month clinical trial, and its open-label extension
Uma Ramaswami, Esperanza Font-Montgomery, Ozlem Goker-Alpan, et al.
JIMD Reports
|
March 17, 2021
Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I
Troy C Lund, Terence M Doherty, Julie B Eisengart, et al.
Communications Medicine
|
January 9, 2025
Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series
Nishitha R Pillai, Ning Liu, Xiyuan Li, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 23, 2009
Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system
Elena L Aronovich, Jason B Bell, Shaukat A Khan, et al.
Journal of Inherited Metabolic Disease
|
December 19, 2023
Quantitative brain morphometry identifies cerebellar, cortical, and subcortical gray and white matter atrophy in late-onset Tay-Sachs disease
Jitka Májovská, Igor Nestrašil, Alia Ahmed, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
March 9, 2018
Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison
Julie B Eisengart, Kyle D Rudser, Yong Xue, et al.
Pharmacoepidemiology and Drug Safety
|
December 16, 2011
Source document verification in the Mucopolysaccharidosis Type I Registry
Karien Verhulst, Laura Artiles-Carloni, Michael Beck, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 28, 2018
Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
Kanut Laoharawee, Russell C DeKelver, Kelly M Podetz-Pedersen, et al.
Molecular Genetics and Metabolism
|
December 17, 2008
Lysosomal Disease Network's "WORLD Symposium 2009". Introduction
Chester B Whitley, John A Barranger, Christine M Eng, et al.
Page
of 12
Search research articles
Search
Showing results (91-100 of 116) with videos related to
Sort By:
Page
of 12
Pediatric Research
|
August 22, 2019
Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation
Lynda E Polgreen, Troy C Lund, Elizabeth Braunlin, et al.
Molecular Genetics and Metabolism
|
April 11, 2025
Safety and efficacy of migalastat in adolescent patients with Fabry disease: Results from ASPIRE, a phase 3b, open-label, single-arm, 12-month clinical trial, and its open-label extension
Uma Ramaswami, Esperanza Font-Montgomery, Ozlem Goker-Alpan, et al.
JIMD Reports
|
March 17, 2021
Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I
Troy C Lund, Terence M Doherty, Julie B Eisengart, et al.
Communications Medicine
|
January 9, 2025
Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series
Nishitha R Pillai, Ning Liu, Xiyuan Li, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 23, 2009
Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system
Elena L Aronovich, Jason B Bell, Shaukat A Khan, et al.
Journal of Inherited Metabolic Disease
|
December 19, 2023
Quantitative brain morphometry identifies cerebellar, cortical, and subcortical gray and white matter atrophy in late-onset Tay-Sachs disease
Jitka Májovská, Igor Nestrašil, Alia Ahmed, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
March 9, 2018
Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison
Julie B Eisengart, Kyle D Rudser, Yong Xue, et al.
Pharmacoepidemiology and Drug Safety
|
December 16, 2011
Source document verification in the Mucopolysaccharidosis Type I Registry
Karien Verhulst, Laura Artiles-Carloni, Michael Beck, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 28, 2018
Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
Kanut Laoharawee, Russell C DeKelver, Kelly M Podetz-Pedersen, et al.
Molecular Genetics and Metabolism
|
December 17, 2008
Lysosomal Disease Network's "WORLD Symposium 2009". Introduction
Chester B Whitley, John A Barranger, Christine M Eng, et al.
Page
of 12