Search research articles
Contact Us
Filters
Showing results (81-90 of 110) with videos related to
Page
of 11
Sort By:
Orphanet Journal of Rare Diseases
|
May 31, 2012
3-methylcrotonyl-CoA carboxylase deficiency: clinical, biochemical, enzymatic and molecular studies in 88 individuals
Sarah C Grünert, Martin Stucki, Raphael J Morscher, et al.
Molecular Genetics and Metabolism
|
February 5, 2018
Complex care of individuals with multiple sulfatase deficiency: Clinical cases and consensus statement
Rebecca Ahrens-Nicklas, Lars Schlotawa, Andrea Ballabio, et al.
Molecular Genetics and Metabolism
|
June 18, 2021
Should patients with Phosphomannomutase 2-CDG (PMM2-CDG) be screened for adrenal insufficiency?
Anna Čechová, Tomáš Honzík, Andrew C Edmondson, et al.
American Journal of Human Genetics
|
December 31, 2005
Epimerase-deficiency galactosemia is not a binary condition
Kimberly K Openo, Jenny M Schulz, Claudia A Vargas, et al.
Clinical Epigenetics
|
April 20, 2022
Epimutations in both the TESK2 and MMACHC promoters in the Epi-cblC inherited disorder of intracellular metabolism of vitamin B<sub>12</sub>
Abderrahim Oussalah, Youssef Siblini, Sébastien Hergalant, et al.
Orphanet Journal of Rare Diseases
|
March 10, 2026
Characterizing the frequency of clinical events and assessing biomarkers in propionic acidemia: a natural history study
Bernd C Schwahn, Gerard T Berry, Hilary J Vernon, et al.
Translational Science of Rare Diseases
|
January 8, 2019
Conducting an investigator-initiated randomized double-blinded intervention trial in acute decompensation of inborn errors of metabolism: Lessons from the N-Carbamylglutamate Consortium
Nicholas Ah Mew, Avital Cnaan, Robert McCarter, et al.
The New England Journal of Medicine
|
May 13, 2026
Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery
Rebecca C Ahrens-Nicklas, Chelsea Kotch, Aoife M Roche, et al.
Frontiers in Genetics
|
August 8, 2022
Population-Based Screening of Newborns: Findings From the NBS Expansion Study (Part One)
Amy Brower, Kee Chan, Marc Williams, et al.
Human Mutation
|
April 14, 2025
Genotype/Phenotype Relationship: Lessons From 137 Patients With PMM2-CDG
Sander Pajusalu, Mari-Anne Vals, Mercedes Serrano, et al.
Page
of 11
Search research articles
Search
Showing results (81-90 of 110) with videos related to
Sort By:
Page
of 11
Orphanet Journal of Rare Diseases
|
May 31, 2012
3-methylcrotonyl-CoA carboxylase deficiency: clinical, biochemical, enzymatic and molecular studies in 88 individuals
Sarah C Grünert, Martin Stucki, Raphael J Morscher, et al.
Molecular Genetics and Metabolism
|
February 5, 2018
Complex care of individuals with multiple sulfatase deficiency: Clinical cases and consensus statement
Rebecca Ahrens-Nicklas, Lars Schlotawa, Andrea Ballabio, et al.
Molecular Genetics and Metabolism
|
June 18, 2021
Should patients with Phosphomannomutase 2-CDG (PMM2-CDG) be screened for adrenal insufficiency?
Anna Čechová, Tomáš Honzík, Andrew C Edmondson, et al.
American Journal of Human Genetics
|
December 31, 2005
Epimerase-deficiency galactosemia is not a binary condition
Kimberly K Openo, Jenny M Schulz, Claudia A Vargas, et al.
Clinical Epigenetics
|
April 20, 2022
Epimutations in both the TESK2 and MMACHC promoters in the Epi-cblC inherited disorder of intracellular metabolism of vitamin B<sub>12</sub>
Abderrahim Oussalah, Youssef Siblini, Sébastien Hergalant, et al.
Orphanet Journal of Rare Diseases
|
March 10, 2026
Characterizing the frequency of clinical events and assessing biomarkers in propionic acidemia: a natural history study
Bernd C Schwahn, Gerard T Berry, Hilary J Vernon, et al.
Translational Science of Rare Diseases
|
January 8, 2019
Conducting an investigator-initiated randomized double-blinded intervention trial in acute decompensation of inborn errors of metabolism: Lessons from the N-Carbamylglutamate Consortium
Nicholas Ah Mew, Avital Cnaan, Robert McCarter, et al.
The New England Journal of Medicine
|
May 13, 2026
Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery
Rebecca C Ahrens-Nicklas, Chelsea Kotch, Aoife M Roche, et al.
Frontiers in Genetics
|
August 8, 2022
Population-Based Screening of Newborns: Findings From the NBS Expansion Study (Part One)
Amy Brower, Kee Chan, Marc Williams, et al.
Human Mutation
|
April 14, 2025
Genotype/Phenotype Relationship: Lessons From 137 Patients With PMM2-CDG
Sander Pajusalu, Mari-Anne Vals, Mercedes Serrano, et al.
Page
of 11