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Journal of Patient-Reported Outcomes
|
October 27, 2025
Signs, symptoms, and health-related quality of life in MELAS: measuring what's important from the patient and clinician perspectives
Paolo Medrano, Benjamin Banderas, Marisa Brimmer, et al.
Human Molecular Genetics
|
March 2, 2022
De novo variants in EMC1 lead to neurodevelopmental delay and cerebellar degeneration and affect glial function in Drosophila
Hyung-Lok Chung, Patrick Rump, Di Lu, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
December 21, 2018
Microdeletions excluding YWHAE and PAFAH1B1 cause a unique leukoencephalopathy: further delineation of the 17p13.3 microdeletion spectrum
Lisa T Emrick, Jill A Rosenfeld, Seema R Lalani, et al.
Cell Reports. Medicine
|
May 31, 2023
Tracer metabolomics reveals the role of aldose reductase in glycosylation
Silvia Radenkovic, Anna N Ligezka, Sneha S Mokashi, et al.
Genes
|
January 21, 2022
Genotype-Phenotype Comparison in POGZ-Related Neurodevelopmental Disorders by Using Clinical Scoring
Dóra Nagy, Sarah Verheyen, Kristen M Wigby, et al.
American Journal of Medical Genetics. Part A
|
January 18, 2023
Insights into the perinatal phenotype of Kabuki syndrome in infants identified by genome-wide sequencing
Kristen Wigby, Monia Hammer, Mari Tokita, et al.
JCI Insight
|
April 8, 2024
A complement C4-derived glycopeptide is a biomarker for PMM2-CDG
Kishore Garapati, Rohit Budhraja, Mayank Saraswat, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
April 29, 2025
Efficacy, safety, and tolerability of chenodeoxycholic acid (CDCA) in adult patients with cerebrotendinous xanthomatosis (RESTORE): A randomized withdrawal, double-blind, placebo-controlled, crossover phase-3 study
Yaz Y Kisanuki, Paulo R Nobrega, Ryan Himes, et al.
Plos Genetics
|
March 29, 2014
Heterozygous de novo and inherited mutations in the smooth muscle actin (ACTG2) gene underlie megacystis-microcolon-intestinal hypoperistalsis syndrome
Michael F Wangler, Claudia Gonzaga-Jauregui, Tomasz Gambin, et al.
Annals of Neurology
|
October 15, 2021
Sorbitol Is a Severity Biomarker for PMM2-CDG with Therapeutic Implications
Anna N Ligezka, Silvia Radenkovic, Mayank Saraswat, et al.
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Search research articles
Search
Showing results (31-40 of 61) with videos related to
Sort By:
Page
of 7
Journal of Patient-Reported Outcomes
|
October 27, 2025
Signs, symptoms, and health-related quality of life in MELAS: measuring what's important from the patient and clinician perspectives
Paolo Medrano, Benjamin Banderas, Marisa Brimmer, et al.
Human Molecular Genetics
|
March 2, 2022
De novo variants in EMC1 lead to neurodevelopmental delay and cerebellar degeneration and affect glial function in Drosophila
Hyung-Lok Chung, Patrick Rump, Di Lu, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
December 21, 2018
Microdeletions excluding YWHAE and PAFAH1B1 cause a unique leukoencephalopathy: further delineation of the 17p13.3 microdeletion spectrum
Lisa T Emrick, Jill A Rosenfeld, Seema R Lalani, et al.
Cell Reports. Medicine
|
May 31, 2023
Tracer metabolomics reveals the role of aldose reductase in glycosylation
Silvia Radenkovic, Anna N Ligezka, Sneha S Mokashi, et al.
Genes
|
January 21, 2022
Genotype-Phenotype Comparison in POGZ-Related Neurodevelopmental Disorders by Using Clinical Scoring
Dóra Nagy, Sarah Verheyen, Kristen M Wigby, et al.
American Journal of Medical Genetics. Part A
|
January 18, 2023
Insights into the perinatal phenotype of Kabuki syndrome in infants identified by genome-wide sequencing
Kristen Wigby, Monia Hammer, Mari Tokita, et al.
JCI Insight
|
April 8, 2024
A complement C4-derived glycopeptide is a biomarker for PMM2-CDG
Kishore Garapati, Rohit Budhraja, Mayank Saraswat, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
April 29, 2025
Efficacy, safety, and tolerability of chenodeoxycholic acid (CDCA) in adult patients with cerebrotendinous xanthomatosis (RESTORE): A randomized withdrawal, double-blind, placebo-controlled, crossover phase-3 study
Yaz Y Kisanuki, Paulo R Nobrega, Ryan Himes, et al.
Plos Genetics
|
March 29, 2014
Heterozygous de novo and inherited mutations in the smooth muscle actin (ACTG2) gene underlie megacystis-microcolon-intestinal hypoperistalsis syndrome
Michael F Wangler, Claudia Gonzaga-Jauregui, Tomasz Gambin, et al.
Annals of Neurology
|
October 15, 2021
Sorbitol Is a Severity Biomarker for PMM2-CDG with Therapeutic Implications
Anna N Ligezka, Silvia Radenkovic, Mayank Saraswat, et al.
Page
of 7