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Neurobiology of Disease
|
March 15, 2011
Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I
Daniel A Wolf, Andrew W Lenander, Zhenhong Nan, et al.
The Journal of Pediatrics
|
May 6, 2004
Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Paul Harmatz, Chester B Whitley, Lewis Waber, et al.
Journal of Clinical Densitometry : the Official Journal of the International Society for Clinical Densitometry
|
April 9, 2013
Low bone mineral content and challenges in interpretation of dual-energy X-ray absorptiometry in children with mucopolysaccharidosis types I, II, and VI
Lynda E Polgreen, William Thomas, Ellen Fung, et al.
Molecular Therapy. Advances
|
May 15, 2026
CSF GAG non-reducing ends in MPS IH after peripheral and brain-penetrating therapies: A model comparing IV non-targeted ERT and HCT
Troy C Lund, John E Wagner, Susie Long, et al.
The Journal of Pediatrics
|
September 15, 2012
Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome
Julie B Eisengart, Kyle D Rudser, Jakub Tolar, et al.
The Journal of Pediatrics
|
January 21, 2016
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA
Elsa G Shapiro, Igor Nestrasil, Kathleen A Delaney, et al.
Journal of Pediatric Rehabilitation Medicine
|
December 31, 2014
Isokinetic muscle strength differences in patients with mucopolysaccharidosis I, II, and VI
Natalie E Taylor, Donald R Dengel, Troy C Lund, et al.
Molecular Genetics and Metabolism
|
July 12, 2021
A multicenter open-label extension study of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A
Frits A Wijburg, Chester B Whitley, Joseph Muenzer, et al.
Journal of Inherited Metabolic Disease
|
February 25, 2023
Persistent bone and joint disease despite current treatments for mucopolysaccharidosis types I, II, and VI: Data from a 10-year prospective study
Bradley S Miller, Ellen B Fung, Klane K White, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 15, 2004
Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene
Seth D Hartung, Joel L Frandsen, Dao Pan, et al.
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of 12
Search research articles
Search
Showing results (61-70 of 116) with videos related to
Sort By:
Page
of 12
Neurobiology of Disease
|
March 15, 2011
Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I
Daniel A Wolf, Andrew W Lenander, Zhenhong Nan, et al.
The Journal of Pediatrics
|
May 6, 2004
Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Paul Harmatz, Chester B Whitley, Lewis Waber, et al.
Journal of Clinical Densitometry : the Official Journal of the International Society for Clinical Densitometry
|
April 9, 2013
Low bone mineral content and challenges in interpretation of dual-energy X-ray absorptiometry in children with mucopolysaccharidosis types I, II, and VI
Lynda E Polgreen, William Thomas, Ellen Fung, et al.
Molecular Therapy. Advances
|
May 15, 2026
CSF GAG non-reducing ends in MPS IH after peripheral and brain-penetrating therapies: A model comparing IV non-targeted ERT and HCT
Troy C Lund, John E Wagner, Susie Long, et al.
The Journal of Pediatrics
|
September 15, 2012
Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome
Julie B Eisengart, Kyle D Rudser, Jakub Tolar, et al.
The Journal of Pediatrics
|
January 21, 2016
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA
Elsa G Shapiro, Igor Nestrasil, Kathleen A Delaney, et al.
Journal of Pediatric Rehabilitation Medicine
|
December 31, 2014
Isokinetic muscle strength differences in patients with mucopolysaccharidosis I, II, and VI
Natalie E Taylor, Donald R Dengel, Troy C Lund, et al.
Molecular Genetics and Metabolism
|
July 12, 2021
A multicenter open-label extension study of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A
Frits A Wijburg, Chester B Whitley, Joseph Muenzer, et al.
Journal of Inherited Metabolic Disease
|
February 25, 2023
Persistent bone and joint disease despite current treatments for mucopolysaccharidosis types I, II, and VI: Data from a 10-year prospective study
Bradley S Miller, Ellen B Fung, Klane K White, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 15, 2004
Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene
Seth D Hartung, Joel L Frandsen, Dao Pan, et al.
Page
of 12