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Molecular Genetics and Metabolism
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June 14, 2025
Differentiation of neonatal and infantile onset ECHS1 deficiency using SCEH enzyme activity and plasma acylcarnitine analysis
Olivia D'Annibale, Whitney Phinney, Molly Crenshaw, et al.
Biorxiv : the Preprint Server for Biology
|
November 24, 2025
Splice-switching antisense oligonucleotides correct cryptic exon inclusion and restore SDCCAG8 protein in Bardet-Biedl Syndrome
Kelleen E McEntee, Bailey L McCurdy, Austin Larson, et al.
Prenatal Diagnosis
|
February 9, 2023
Piloting a multidisciplinary approach to improve outcomes of fetal whole exome sequencing: An overview of workflow and case example
Kestutis C Micke, Hannah M Elfman, Katherine A Fantauzzo, et al.
Molecular Genetics and Metabolism
|
January 13, 2026
L-fucose supplementation in a patient with global hypofucosylation and a mono-allelic variant in SLC35C1: Clinical improvement and assessment of biomarkers
Rodrigo T Starosta, Miao He, Sara Gracie, et al.
Neurology
|
February 20, 2025
Adult-onset Leigh Syndrome: An analysis of the North American Mitochondrial Disease Consortium Database (P3-11.017)
Emanuele Barca, Adam Kroopnick, Alexander Houck, et al.
American Journal of Medical Genetics. Part A
|
November 6, 2024
Expanding the Molecular and Clinical Phenotype of Patients With De Novo Variants in KIF5C: A Six Patient Case Series
Sara Gracie, Prasannakumar Deshpande, Patrik Hollos, et al.
Molecular Genetics and Metabolism
|
March 23, 2025
Goal attainment in PMM2-CDG: A new approach measuring meaningful clinical outcomes
Sanne Verberkmoes, Gina L Mazza, Andrew C Edmondson, et al.
Molecular Genetics and Metabolism
|
November 12, 2017
New insights into the phenotype of FARS2 deficiency
Elise Vantroys, Austin Larson, Marisa Friederich, et al.
Frontiers in Pediatrics
|
March 5, 2024
Multi-center implementation of rapid whole genome sequencing provides additional evidence of its utility in the pediatric inpatient setting
Lauren Thompson, Austin Larson, Lisa Salz, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 15, 2023
A new neurodevelopmental disorder linked to heterozygous variants in UNC79
Allan Bayat, Zhenjiang Liu, Sheng Luo, et al.
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of 7
Search research articles
Search
Showing results (21-30 of 61) with videos related to
Sort By:
Page
of 7
Molecular Genetics and Metabolism
|
June 14, 2025
Differentiation of neonatal and infantile onset ECHS1 deficiency using SCEH enzyme activity and plasma acylcarnitine analysis
Olivia D'Annibale, Whitney Phinney, Molly Crenshaw, et al.
Biorxiv : the Preprint Server for Biology
|
November 24, 2025
Splice-switching antisense oligonucleotides correct cryptic exon inclusion and restore SDCCAG8 protein in Bardet-Biedl Syndrome
Kelleen E McEntee, Bailey L McCurdy, Austin Larson, et al.
Prenatal Diagnosis
|
February 9, 2023
Piloting a multidisciplinary approach to improve outcomes of fetal whole exome sequencing: An overview of workflow and case example
Kestutis C Micke, Hannah M Elfman, Katherine A Fantauzzo, et al.
Molecular Genetics and Metabolism
|
January 13, 2026
L-fucose supplementation in a patient with global hypofucosylation and a mono-allelic variant in SLC35C1: Clinical improvement and assessment of biomarkers
Rodrigo T Starosta, Miao He, Sara Gracie, et al.
Neurology
|
February 20, 2025
Adult-onset Leigh Syndrome: An analysis of the North American Mitochondrial Disease Consortium Database (P3-11.017)
Emanuele Barca, Adam Kroopnick, Alexander Houck, et al.
American Journal of Medical Genetics. Part A
|
November 6, 2024
Expanding the Molecular and Clinical Phenotype of Patients With De Novo Variants in KIF5C: A Six Patient Case Series
Sara Gracie, Prasannakumar Deshpande, Patrik Hollos, et al.
Molecular Genetics and Metabolism
|
March 23, 2025
Goal attainment in PMM2-CDG: A new approach measuring meaningful clinical outcomes
Sanne Verberkmoes, Gina L Mazza, Andrew C Edmondson, et al.
Molecular Genetics and Metabolism
|
November 12, 2017
New insights into the phenotype of FARS2 deficiency
Elise Vantroys, Austin Larson, Marisa Friederich, et al.
Frontiers in Pediatrics
|
March 5, 2024
Multi-center implementation of rapid whole genome sequencing provides additional evidence of its utility in the pediatric inpatient setting
Lauren Thompson, Austin Larson, Lisa Salz, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 15, 2023
A new neurodevelopmental disorder linked to heterozygous variants in UNC79
Allan Bayat, Zhenjiang Liu, Sheng Luo, et al.
Page
of 7