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Neurology. Genetics
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December 25, 2024
Clinical Heterogeneity of Neuronal Ceroid Lipofuscinosis Type 13: A Case Report and Systematic Review of Literature
Nikhil B Ghayal, Shanu F Roemer, Philip W Tipton, et al.
Journal of Inherited Metabolic Disease
|
September 26, 2025
Screening and Diagnosis of Lysosomal Disorders: Biochemical and Genomic Approaches
Matthew J Schultz, Patricia L Hall, Gisele Bentz Pino, et al.
Molecular Genetics and Metabolism
|
August 31, 2024
Sensitivity of transferrin isoform analysis for PMM2-CDG
Patrica L Hall, Kris Liedke, Coleman Turgeon, et al.
Molecular Genetics and Metabolism Reports
|
July 18, 2024
Pre-analytic decrease of phenylalanine in plasma of patients with phenylketonuria treated with pegvaliase
Coleman Turgeon, Kari Casas, Ryan Flanagan, et al.
The EMBO Journal
|
July 21, 2016
Natural underlying mtDNA heteroplasmy as a potential source of intra-person hiPSC variability
Ester Perales-Clemente, Alexandra N Cook, Jared M Evans, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
August 4, 2025
Rethinking the pathogenicity of intragenic DMD duplications detected by carrier screening: High prevalence of nontandem duplications revealed by long-read sequencing
Qiliang Ding, Jagadheshwar Balan, Noemi Vidal-Folch, et al.
Molecular Genetics and Metabolism
|
November 1, 2025
Clinical utility of untargeted urine oligosaccharide screening
Gisele Bentz Pino, Marie A Quade, Matthew J Schultz, et al.
Neuromuscular Disorders : NMD
|
December 25, 2025
Clinically discordant siblings with spinal muscular atrophy: insights from their patient-specific iPSC-derived motor neurons and literature review
Sara L Cook, Tyller Mensa, Henry Noma, et al.
Journal of Inherited Metabolic Disease
|
March 13, 2015
Measurement of psychosine in dried blood spots--a possible improvement to newborn screening programs for Krabbe disease
Coleman T Turgeon, Joseph J Orsini, Karen A Sanders, et al.
Molecular Genetics and Metabolism
|
November 23, 2019
Multiplex testing for the screening of lysosomal storage disease in urine: Sulfatides and glycosaminoglycan profiles in 40 cases of sulfatiduria
Gisele Pino, Erin Conboy, Silvia Tortorelli, et al.
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of 10
Search research articles
Search
Showing results (61-70 of 99) with videos related to
Sort By:
Page
of 10
Neurology. Genetics
|
December 25, 2024
Clinical Heterogeneity of Neuronal Ceroid Lipofuscinosis Type 13: A Case Report and Systematic Review of Literature
Nikhil B Ghayal, Shanu F Roemer, Philip W Tipton, et al.
Journal of Inherited Metabolic Disease
|
September 26, 2025
Screening and Diagnosis of Lysosomal Disorders: Biochemical and Genomic Approaches
Matthew J Schultz, Patricia L Hall, Gisele Bentz Pino, et al.
Molecular Genetics and Metabolism
|
August 31, 2024
Sensitivity of transferrin isoform analysis for PMM2-CDG
Patrica L Hall, Kris Liedke, Coleman Turgeon, et al.
Molecular Genetics and Metabolism Reports
|
July 18, 2024
Pre-analytic decrease of phenylalanine in plasma of patients with phenylketonuria treated with pegvaliase
Coleman Turgeon, Kari Casas, Ryan Flanagan, et al.
The EMBO Journal
|
July 21, 2016
Natural underlying mtDNA heteroplasmy as a potential source of intra-person hiPSC variability
Ester Perales-Clemente, Alexandra N Cook, Jared M Evans, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
August 4, 2025
Rethinking the pathogenicity of intragenic DMD duplications detected by carrier screening: High prevalence of nontandem duplications revealed by long-read sequencing
Qiliang Ding, Jagadheshwar Balan, Noemi Vidal-Folch, et al.
Molecular Genetics and Metabolism
|
November 1, 2025
Clinical utility of untargeted urine oligosaccharide screening
Gisele Bentz Pino, Marie A Quade, Matthew J Schultz, et al.
Neuromuscular Disorders : NMD
|
December 25, 2025
Clinically discordant siblings with spinal muscular atrophy: insights from their patient-specific iPSC-derived motor neurons and literature review
Sara L Cook, Tyller Mensa, Henry Noma, et al.
Journal of Inherited Metabolic Disease
|
March 13, 2015
Measurement of psychosine in dried blood spots--a possible improvement to newborn screening programs for Krabbe disease
Coleman T Turgeon, Joseph J Orsini, Karen A Sanders, et al.
Molecular Genetics and Metabolism
|
November 23, 2019
Multiplex testing for the screening of lysosomal storage disease in urine: Sulfatides and glycosaminoglycan profiles in 40 cases of sulfatiduria
Gisele Pino, Erin Conboy, Silvia Tortorelli, et al.
Page
of 10