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Molecular Genetics and Metabolism
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November 29, 2008
A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I
Roberto Giugliani, Verónica Muñoz Rojas, Ana Maria Martins, et al.
Pediatrics
|
July 16, 2008
A prospective, cross-sectional survey study of the natural history of Niemann-Pick disease type B
Margaret M McGovern, Melissa P Wasserstein, Roberto Giugliani, et al.
Pediatrics
|
October 4, 2006
Factors associated with establishing a causal diagnosis for children with cardiomyopathy
Gerald F Cox, Lynn A Sleeper, April M Lowe, et al.
American Heart Journal
|
June 3, 2008
Characteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy: a comparative study from the Pediatric Cardiomyopathy Registry
David M Connuck, Lynn A Sleeper, Steven D Colan, et al.
Heart Failure Clinics
|
September 28, 2010
The pediatric cardiomyopathy registry and heart failure: key results from the first 15 years
James D Wilkinson, David C Landy, Steven D Colan, et al.
The New England Journal of Medicine
|
April 25, 2003
The incidence of pediatric cardiomyopathy in two regions of the United States
Steven E Lipshultz, Lynn A Sleeper, Jeffrey A Towbin, et al.
Biomedical Journal
|
September 23, 2023
CAN008 prolongs overall survival in patients with newly diagnosed GBM characterized by high tumor mutational burden
Ian Yi-Feng Chang, Hong-Chieh Tsai, Chia-Hua Chen, et al.
JAMA
|
October 19, 2006
Incidence, causes, and outcomes of dilated cardiomyopathy in children
Jeffrey A Towbin, April M Lowe, Steven D Colan, et al.
Lancet (London, England)
|
September 10, 2013
Risk stratification at diagnosis for children with hypertrophic cardiomyopathy: an analysis of data from the Pediatric Cardiomyopathy Registry
Steven E Lipshultz, E John Orav, James D Wilkinson, et al.
EMBO Molecular Medicine
|
February 27, 2024
Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor
Qing Xie, Xiupeng Chen, Hong Ma, et al.
Page
of 8
Search research articles
Search
Showing results (51-60 of 75) with videos related to
Sort By:
Page
of 8
Molecular Genetics and Metabolism
|
November 29, 2008
A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I
Roberto Giugliani, Verónica Muñoz Rojas, Ana Maria Martins, et al.
Pediatrics
|
July 16, 2008
A prospective, cross-sectional survey study of the natural history of Niemann-Pick disease type B
Margaret M McGovern, Melissa P Wasserstein, Roberto Giugliani, et al.
Pediatrics
|
October 4, 2006
Factors associated with establishing a causal diagnosis for children with cardiomyopathy
Gerald F Cox, Lynn A Sleeper, April M Lowe, et al.
American Heart Journal
|
June 3, 2008
Characteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy: a comparative study from the Pediatric Cardiomyopathy Registry
David M Connuck, Lynn A Sleeper, Steven D Colan, et al.
Heart Failure Clinics
|
September 28, 2010
The pediatric cardiomyopathy registry and heart failure: key results from the first 15 years
James D Wilkinson, David C Landy, Steven D Colan, et al.
The New England Journal of Medicine
|
April 25, 2003
The incidence of pediatric cardiomyopathy in two regions of the United States
Steven E Lipshultz, Lynn A Sleeper, Jeffrey A Towbin, et al.
Biomedical Journal
|
September 23, 2023
CAN008 prolongs overall survival in patients with newly diagnosed GBM characterized by high tumor mutational burden
Ian Yi-Feng Chang, Hong-Chieh Tsai, Chia-Hua Chen, et al.
JAMA
|
October 19, 2006
Incidence, causes, and outcomes of dilated cardiomyopathy in children
Jeffrey A Towbin, April M Lowe, Steven D Colan, et al.
Lancet (London, England)
|
September 10, 2013
Risk stratification at diagnosis for children with hypertrophic cardiomyopathy: an analysis of data from the Pediatric Cardiomyopathy Registry
Steven E Lipshultz, E John Orav, James D Wilkinson, et al.
EMBO Molecular Medicine
|
February 27, 2024
Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor
Qing Xie, Xiupeng Chen, Hong Ma, et al.
Page
of 8