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Chester B Whitley

Showing results (101-110 of 116) with videos related to

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Neurology|April 14, 2019
Attention and corpus callosum volumes in individuals with mucopolysaccharidosis type IKelly E King, Kyle D Rudser, Igor Nestrasil, et al.
Molecular Therapy. Methods & Clinical Development|December 17, 2024
Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS ILalitha R Belur, Avery K Huber, Hillary Mantone, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|February 17, 2005
Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndromeSatkiran S Grewal, Robert Wynn, Jose E Abdenur, et al.
Journal of Pediatric Gastroenterology and Nutrition|August 8, 2015
Clinical Features of Lysosomal Acid Lipase DeficiencyBarbara K Burton, Patrick B Deegan, Gregory M Enns, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 12, 2018
ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler SyndromeLi Ou, Russell C DeKelver, Michelle Rohde, et al.
Molecular Genetics and Metabolism|May 27, 2008
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatasePaul Harmatz, Roberto Giugliani, Ida Vanessa D Schwartz, et al.
Journal of Pediatric Rehabilitation Medicine|July 17, 2010
Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfataseCeleste Decker, Zi-Fan Yu, Roberto Giugliani, et al.
Journal of Inherited Metabolic Disease|February 9, 2010
Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatasePaul Harmatz, Zi-Fan Yu, Roberto Giugliani, et al.
Molecular Genetics and Metabolism|June 23, 2015
Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatmentElsa G Shapiro, Igor Nestrasil, Kyle Rudser, et al.
The Journal of Pediatrics|March 6, 2009
Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled studyFriedrich K Trefz, Barbara K Burton, Nicola Longo, et al.
Pageof 12

Showing results (101-110 of 116) with videos related to

Sort By:
Pageof 12
Neurology|April 14, 2019
Attention and corpus callosum volumes in individuals with mucopolysaccharidosis type IKelly E King, Kyle D Rudser, Igor Nestrasil, et al.
Molecular Therapy. Methods & Clinical Development|December 17, 2024
Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS ILalitha R Belur, Avery K Huber, Hillary Mantone, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|February 17, 2005
Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndromeSatkiran S Grewal, Robert Wynn, Jose E Abdenur, et al.
Journal of Pediatric Gastroenterology and Nutrition|August 8, 2015
Clinical Features of Lysosomal Acid Lipase DeficiencyBarbara K Burton, Patrick B Deegan, Gregory M Enns, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 12, 2018
ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler SyndromeLi Ou, Russell C DeKelver, Michelle Rohde, et al.
Molecular Genetics and Metabolism|May 27, 2008
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatasePaul Harmatz, Roberto Giugliani, Ida Vanessa D Schwartz, et al.
Journal of Pediatric Rehabilitation Medicine|July 17, 2010
Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfataseCeleste Decker, Zi-Fan Yu, Roberto Giugliani, et al.
Journal of Inherited Metabolic Disease|February 9, 2010
Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatasePaul Harmatz, Zi-Fan Yu, Roberto Giugliani, et al.
Molecular Genetics and Metabolism|June 23, 2015
Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatmentElsa G Shapiro, Igor Nestrasil, Kyle Rudser, et al.
The Journal of Pediatrics|March 6, 2009
Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled studyFriedrich K Trefz, Barbara K Burton, Nicola Longo, et al.
Pageof 12