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Molecular Genetics and Metabolism
|
August 12, 2022
Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II
Joseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
Neurology
|
June 13, 2025
Disease-Modifying, Neuroprotective Effect of N-Acetyl-l-Leucine in Adult and Pediatric Patients With Niemann-Pick Disease Type C
Marc C Patterson, Uma Ramaswami, Aimee Donald, et al.
Journal of Inherited Metabolic Disease
|
May 28, 2021
High dose genistein in Sanfilippo syndrome: A randomised controlled trial
Arunabha Ghosh, Stewart Rust, Kia Langford-Smith, et al.
Molecular Genetics and Metabolism
|
August 26, 2022
Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study
Joseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
Molecular Therapy. Methods & Clinical Development
|
May 3, 2019
Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated <i>Ex Vivo</i> Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA
Stuart M Ellison, Aiyin Liao, Shaun Wood, et al.
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation
|
February 25, 2015
Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines
Mieke Aldenhoven, Simon A Jones, Denise Bonney, et al.
Blood Cell Therapy
|
October 27, 2023
A decade of excellent transplant survival in children with inherited metabolic diseases: A report from a single metabolic transplant centre in Europe
Su Han Lum, Andrew Will, Heather J Church, et al.
American Journal of Medical Genetics. Part A
|
June 13, 2015
Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study
Barbara K Burton, Kenneth I Berger, Gregory D Lewis, et al.
Molecular Genetics and Metabolism
|
May 27, 2025
Newborn screening for metachromatic leukodystrophy: Preparation of reagents and methodology for measurement of sulfatides and arylsulfatase A enzymatic activity in dried blood spots
Austin Shaff, Khaja Basheeruddin, Soumeya Bekri, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 11, 2013
Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease
Ana Sergijenko, Alexander Langford-Smith, Ai Y Liao, et al.
Page
of 26
Search research articles
Search
Showing results (191-200 of 259) with videos related to
Sort By:
Page
of 26
Molecular Genetics and Metabolism
|
August 12, 2022
Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II
Joseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
Neurology
|
June 13, 2025
Disease-Modifying, Neuroprotective Effect of N-Acetyl-l-Leucine in Adult and Pediatric Patients With Niemann-Pick Disease Type C
Marc C Patterson, Uma Ramaswami, Aimee Donald, et al.
Journal of Inherited Metabolic Disease
|
May 28, 2021
High dose genistein in Sanfilippo syndrome: A randomised controlled trial
Arunabha Ghosh, Stewart Rust, Kia Langford-Smith, et al.
Molecular Genetics and Metabolism
|
August 26, 2022
Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study
Joseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
Molecular Therapy. Methods & Clinical Development
|
May 3, 2019
Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated <i>Ex Vivo</i> Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA
Stuart M Ellison, Aiyin Liao, Shaun Wood, et al.
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation
|
February 25, 2015
Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines
Mieke Aldenhoven, Simon A Jones, Denise Bonney, et al.
Blood Cell Therapy
|
October 27, 2023
A decade of excellent transplant survival in children with inherited metabolic diseases: A report from a single metabolic transplant centre in Europe
Su Han Lum, Andrew Will, Heather J Church, et al.
American Journal of Medical Genetics. Part A
|
June 13, 2015
Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study
Barbara K Burton, Kenneth I Berger, Gregory D Lewis, et al.
Molecular Genetics and Metabolism
|
May 27, 2025
Newborn screening for metachromatic leukodystrophy: Preparation of reagents and methodology for measurement of sulfatides and arylsulfatase A enzymatic activity in dried blood spots
Austin Shaff, Khaja Basheeruddin, Soumeya Bekri, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 11, 2013
Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease
Ana Sergijenko, Alexander Langford-Smith, Ai Y Liao, et al.
Page
of 26