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John J Mitchell

Showing results (41-50 of 73) with videos related to

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Molecular Genetics and Metabolism|July 7, 2016
Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndromeChristian J Hendriksz, Rossella Parini, Moeenaldeen D AlSayed, et al.
Molecular Genetics and Metabolism|January 14, 2015
Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndromePaul R Harmatz, Karl Eugen Mengel, Roberto Giugliani, et al.
The New England Journal of Medicine|December 30, 2025
An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis IIJoseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
Trials|November 18, 2021
Establishing a core outcome set for mucopolysaccharidoses (MPS) in children: study protocol for a rapid literature review, candidate outcomes survey, and Delphi surveysAlison H Howie, Kylie Tingley, Michal Inbar-Feigenberg, et al.
Journal of Inherited Metabolic Disease|July 26, 2015
Child and family experiences with inborn errors of metabolism: a qualitative interview study with representatives of patient groupsSara D Khangura, Kylie Tingley, Pranesh Chakraborty, et al.
Med (New York, N.Y.)|November 15, 2024
Multi-year enzyme expression in patients with mucopolysaccharidosis type VI after liver-directed gene therapyAlessandro Rossi, Roberta Romano, Simona Fecarotta, et al.
Trials|December 10, 2021
Correction to: Establishing a core outcome set for mucopolysaccharidoses (MPS) in children: study protocol for a rapid literature review, candidate outcomes survey, and Delphi surveysAlison H Howie, Kylie Tingley, Michal Inbar-Feigenberg, et al.
The Journal of Clinical Investigation|August 24, 2023
Peroxisome disruption alters lipid metabolism and potentiates antitumor response with MAPK-targeted therapy in melanomaFan Huang, Feiyang Cai, Michael S Dahabieh, et al.
BMC Pediatrics|April 18, 2015
Scoping review of patient- and family-oriented outcomes and measures for chronic pediatric diseaseSara D Khangura, Maria D Karaceper, Yannis Trakadis, et al.
Trials|December 21, 2017
Establishing core outcome sets for phenylketonuria (PKU) and medium-chain Acyl-CoA dehydrogenase (MCAD) deficiency in children: study protocol for systematic reviews and Delphi surveysBeth K Potter, Brian Hutton, Tammy J Clifford, et al.
Pageof 8

Showing results (41-50 of 73) with videos related to

Sort By:
Pageof 8
Molecular Genetics and Metabolism|July 7, 2016
Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndromeChristian J Hendriksz, Rossella Parini, Moeenaldeen D AlSayed, et al.
Molecular Genetics and Metabolism|January 14, 2015
Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndromePaul R Harmatz, Karl Eugen Mengel, Roberto Giugliani, et al.
The New England Journal of Medicine|December 30, 2025
An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis IIJoseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
Trials|November 18, 2021
Establishing a core outcome set for mucopolysaccharidoses (MPS) in children: study protocol for a rapid literature review, candidate outcomes survey, and Delphi surveysAlison H Howie, Kylie Tingley, Michal Inbar-Feigenberg, et al.
Journal of Inherited Metabolic Disease|July 26, 2015
Child and family experiences with inborn errors of metabolism: a qualitative interview study with representatives of patient groupsSara D Khangura, Kylie Tingley, Pranesh Chakraborty, et al.
Med (New York, N.Y.)|November 15, 2024
Multi-year enzyme expression in patients with mucopolysaccharidosis type VI after liver-directed gene therapyAlessandro Rossi, Roberta Romano, Simona Fecarotta, et al.
Trials|December 10, 2021
Correction to: Establishing a core outcome set for mucopolysaccharidoses (MPS) in children: study protocol for a rapid literature review, candidate outcomes survey, and Delphi surveysAlison H Howie, Kylie Tingley, Michal Inbar-Feigenberg, et al.
The Journal of Clinical Investigation|August 24, 2023
Peroxisome disruption alters lipid metabolism and potentiates antitumor response with MAPK-targeted therapy in melanomaFan Huang, Feiyang Cai, Michael S Dahabieh, et al.
BMC Pediatrics|April 18, 2015
Scoping review of patient- and family-oriented outcomes and measures for chronic pediatric diseaseSara D Khangura, Maria D Karaceper, Yannis Trakadis, et al.
Trials|December 21, 2017
Establishing core outcome sets for phenylketonuria (PKU) and medium-chain Acyl-CoA dehydrogenase (MCAD) deficiency in children: study protocol for systematic reviews and Delphi surveysBeth K Potter, Brian Hutton, Tammy J Clifford, et al.
Pageof 8