Search research articles
Contact Us
Filters
Showing results (11-20 of 61) with videos related to
Page
of 7
Sort By:
JIMD Reports
|
November 19, 2016
Cerebrotendinous Xanthomatosis Presenting with Infantile Spasms and Intellectual Disability
Austin Larson, James D Weisfeld-Adams, Tim A Benke, et al.
Pediatric Dermatology
|
March 9, 2026
Neonatal KLHL24-Associated Epidermolysis Bullosa Simplex: Clinical Presentation and Genetic Confirmation of a Rare Skin Fragility Syndrome
Sofia Guelfand Warnken, Jon Kibbie, Austin Larson, et al.
Advances in Therapy
|
December 19, 2023
Living with Cerebrotendinous Xanthomatosis: Patient, Caregiver, and Expert Perspectives
Robert D Steiner, Andrea DeBarber, Austin Larson, et al.
Molecular Genetics and Metabolism
|
February 9, 2025
Causes of mortality in the congenital disorders of glycosylation
Hana Alharbi, Seishu Horikoshi, Sabrina Malone Jenkins, et al.
Molecular Genetics and Metabolism
|
August 30, 2023
Defining the phenotype of PGAP3-congenital disorder of glycosylation; a review of 65 cases
Ruqaiah Altassan, Michael M Allers, Diederik De Graef, et al.
Journal of Clinical Lipidology
|
April 18, 2024
Genetically and clinically confirmed atypical cerebrotendinous xanthomatosis with normal cholestanol and marked elevations of bile acid precursors and bile alcohols
Andrea E DeBarber, Ernst J Schaefer, Jenny Do, et al.
American Journal of Medical Genetics. Part A
|
February 28, 2022
De novo loss-of-function variant in PTDSS1 is associated with developmental delay
Sara Gracie, Nivedita Sengupta, Carlos Ferreira, et al.
Cold Spring Harbor Molecular Case Studies
|
May 22, 2021
Neurodevelopmental phenotypes in individuals with pathogenic variants in <i>CHAMP1</i>
Madison Garrity, Haluk Kavus, Marta Rojas-Vasquez, et al.
American Journal of Medical Genetics. Part A
|
January 3, 2024
Novel molecular mechanism in Malan syndrome uncovered through genome sequencing reanalysis, exon-level Array, and RNA sequencing
Jian Zhao, Nicola Longo, Robert G Lewis, et al.
Molecular Genetics and Metabolism
|
June 25, 2024
Cardiomyopathy, an uncommon phenotype of congenital disorders of glycosylation: Recommendations for baseline screening and follow-up evaluation
Roni Zemet, Kyle D Hope, Andrew C Edmondson, et al.
Page
of 7
Search research articles
Search
Showing results (11-20 of 61) with videos related to
Sort By:
Page
of 7
JIMD Reports
|
November 19, 2016
Cerebrotendinous Xanthomatosis Presenting with Infantile Spasms and Intellectual Disability
Austin Larson, James D Weisfeld-Adams, Tim A Benke, et al.
Pediatric Dermatology
|
March 9, 2026
Neonatal KLHL24-Associated Epidermolysis Bullosa Simplex: Clinical Presentation and Genetic Confirmation of a Rare Skin Fragility Syndrome
Sofia Guelfand Warnken, Jon Kibbie, Austin Larson, et al.
Advances in Therapy
|
December 19, 2023
Living with Cerebrotendinous Xanthomatosis: Patient, Caregiver, and Expert Perspectives
Robert D Steiner, Andrea DeBarber, Austin Larson, et al.
Molecular Genetics and Metabolism
|
February 9, 2025
Causes of mortality in the congenital disorders of glycosylation
Hana Alharbi, Seishu Horikoshi, Sabrina Malone Jenkins, et al.
Molecular Genetics and Metabolism
|
August 30, 2023
Defining the phenotype of PGAP3-congenital disorder of glycosylation; a review of 65 cases
Ruqaiah Altassan, Michael M Allers, Diederik De Graef, et al.
Journal of Clinical Lipidology
|
April 18, 2024
Genetically and clinically confirmed atypical cerebrotendinous xanthomatosis with normal cholestanol and marked elevations of bile acid precursors and bile alcohols
Andrea E DeBarber, Ernst J Schaefer, Jenny Do, et al.
American Journal of Medical Genetics. Part A
|
February 28, 2022
De novo loss-of-function variant in PTDSS1 is associated with developmental delay
Sara Gracie, Nivedita Sengupta, Carlos Ferreira, et al.
Cold Spring Harbor Molecular Case Studies
|
May 22, 2021
Neurodevelopmental phenotypes in individuals with pathogenic variants in <i>CHAMP1</i>
Madison Garrity, Haluk Kavus, Marta Rojas-Vasquez, et al.
American Journal of Medical Genetics. Part A
|
January 3, 2024
Novel molecular mechanism in Malan syndrome uncovered through genome sequencing reanalysis, exon-level Array, and RNA sequencing
Jian Zhao, Nicola Longo, Robert G Lewis, et al.
Molecular Genetics and Metabolism
|
June 25, 2024
Cardiomyopathy, an uncommon phenotype of congenital disorders of glycosylation: Recommendations for baseline screening and follow-up evaluation
Roni Zemet, Kyle D Hope, Andrew C Edmondson, et al.
Page
of 7