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J Edmond Wraith

Showing results (11-20 of 23) with videos related to

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Journal of Inherited Metabolic Disease|January 22, 2011
Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H)Chris J Gardner, Nicola Robinson, Tim Meadows, et al.
JIMD Reports|December 8, 2017
Haematopoietic Stem Cell Transplantation Arrests the Progression of Neurodegenerative Disease in Late-Onset Tay-Sachs DiseaseKarolina M Stepien, Su Han Lum, J Edmond Wraith, et al.
European Journal of Pediatrics|November 27, 2007
Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapyJ Edmond Wraith, Maurizio Scarpa, Michael Beck, et al.
The Journal of Biological Chemistry|November 1, 2014
Heparan sulfate inhibits hematopoietic stem and progenitor cell migration and engraftment in mucopolysaccharidosis IH Angharad Watson, Rebecca J Holley, Kia J Langford-Smith, et al.
Pediatrics|January 2, 2009
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis ILorne A Clarke, J Edmond Wraith, Michael Beck, et al.
Molecular Genetics and Metabolism|March 6, 2007
The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type IGregory M Pastores, Pamela Arn, Michael Beck, 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 diseaseAna Sergijenko, Alexander Langford-Smith, Ai Y Liao, et al.
Hepatology (Baltimore, Md.)|January 26, 2013
Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage diseaseManisha Balwani, Catherine Breen, Gregory M Enns, et al.
The Journal of Pediatrics|May 2, 2006
Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension studyPaul Harmatz, Roberto Giugliani, Ida Schwartz, 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.
Pageof 3

Showing results (11-20 of 23) with videos related to

Sort By:
Pageof 3
Journal of Inherited Metabolic Disease|January 22, 2011
Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H)Chris J Gardner, Nicola Robinson, Tim Meadows, et al.
JIMD Reports|December 8, 2017
Haematopoietic Stem Cell Transplantation Arrests the Progression of Neurodegenerative Disease in Late-Onset Tay-Sachs DiseaseKarolina M Stepien, Su Han Lum, J Edmond Wraith, et al.
European Journal of Pediatrics|November 27, 2007
Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapyJ Edmond Wraith, Maurizio Scarpa, Michael Beck, et al.
The Journal of Biological Chemistry|November 1, 2014
Heparan sulfate inhibits hematopoietic stem and progenitor cell migration and engraftment in mucopolysaccharidosis IH Angharad Watson, Rebecca J Holley, Kia J Langford-Smith, et al.
Pediatrics|January 2, 2009
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis ILorne A Clarke, J Edmond Wraith, Michael Beck, et al.
Molecular Genetics and Metabolism|March 6, 2007
The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type IGregory M Pastores, Pamela Arn, Michael Beck, 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 diseaseAna Sergijenko, Alexander Langford-Smith, Ai Y Liao, et al.
Hepatology (Baltimore, Md.)|January 26, 2013
Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage diseaseManisha Balwani, Catherine Breen, Gregory M Enns, et al.
The Journal of Pediatrics|May 2, 2006
Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension studyPaul Harmatz, Roberto Giugliani, Ida Schwartz, 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.
Pageof 3