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Neurology
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October 23, 2020
Phenylalanine Effects on Brain Function in Adult Phenylketonuria
Andrea Pilotto, Carl M Zipser, Edytha Leks, et al.
Orphanet Journal of Rare Diseases
|
October 21, 2021
<sup>1</sup>H-NMR-based metabolic profiling identifies non-invasive diagnostic and predictive urinary fingerprints in 5q spinal muscular atrophy
Afshin Saffari, Claire Cannet, Astrid Blaschek, et al.
Annals of Neurology
|
April 25, 2019
Impact of Diagnosis and Therapy on Cognitive Function in Urea Cycle Disorders
Roland Posset, Andrea L Gropman, Sandesh C S Nagamani, et al.
American Journal of Human Genetics
|
January 26, 2016
Bi-allelic Truncating Mutations in TANGO2 Cause Infancy-Onset Recurrent Metabolic Crises with Encephalocardiomyopathy
Laura S Kremer, Felix Distelmaier, Bader Alhaddad, et al.
Journal of Inherited Metabolic Disease
|
March 25, 2011
Diagnosis and management of glutaric aciduria type I--revised recommendations
Stefan Kölker, Ernst Christensen, James V Leonard, et al.
Orphanet Journal of Rare Diseases
|
January 20, 2017
Consensus guideline for the diagnosis and treatment of aromatic l-amino acid decarboxylase (AADC) deficiency
Tessa Wassenberg, Marta Molero-Luis, Kathrin Jeltsch, et al.
Aging
|
November 8, 2015
A novel autosomal recessive TERT T1129P mutation in a dyskeratosis congenita family leads to cellular senescence and loss of CD34+ hematopoietic stem cells not reversible by mTOR-inhibition
Clemens Stockklausner, Simon Raffel, Julia Klermund, et al.
Journal of Inherited Metabolic Disease
|
November 18, 2016
Proposed recommendations for diagnosing and managing individuals with glutaric aciduria type I: second revision
Nikolas Boy, Chris Mühlhausen, Esther M Maier, et al.
European Journal of Human Genetics : EJHG
|
January 19, 2018
Compound heterozygous SPATA5 variants in four families and functional studies of SPATA5 deficiency
Sanna Puusepp, Reka Kovacs-Nagy, Bader Alhaddad, et al.
Journal of Inherited Metabolic Disease
|
June 8, 2026
Prevalence, Disease Onset and Clinical Outcome in Arginase 1 Deficiency: Cross-Border Surveillance in Germany, Austria, and Switzerland
Svenja Scharre, Annette L Hess, Florian Gleich, et al.
Page
of 39
Search research articles
Search
Showing results (301-310 of 381) with videos related to
Sort By:
Page
of 39
Neurology
|
October 23, 2020
Phenylalanine Effects on Brain Function in Adult Phenylketonuria
Andrea Pilotto, Carl M Zipser, Edytha Leks, et al.
Orphanet Journal of Rare Diseases
|
October 21, 2021
<sup>1</sup>H-NMR-based metabolic profiling identifies non-invasive diagnostic and predictive urinary fingerprints in 5q spinal muscular atrophy
Afshin Saffari, Claire Cannet, Astrid Blaschek, et al.
Annals of Neurology
|
April 25, 2019
Impact of Diagnosis and Therapy on Cognitive Function in Urea Cycle Disorders
Roland Posset, Andrea L Gropman, Sandesh C S Nagamani, et al.
American Journal of Human Genetics
|
January 26, 2016
Bi-allelic Truncating Mutations in TANGO2 Cause Infancy-Onset Recurrent Metabolic Crises with Encephalocardiomyopathy
Laura S Kremer, Felix Distelmaier, Bader Alhaddad, et al.
Journal of Inherited Metabolic Disease
|
March 25, 2011
Diagnosis and management of glutaric aciduria type I--revised recommendations
Stefan Kölker, Ernst Christensen, James V Leonard, et al.
Orphanet Journal of Rare Diseases
|
January 20, 2017
Consensus guideline for the diagnosis and treatment of aromatic l-amino acid decarboxylase (AADC) deficiency
Tessa Wassenberg, Marta Molero-Luis, Kathrin Jeltsch, et al.
Aging
|
November 8, 2015
A novel autosomal recessive TERT T1129P mutation in a dyskeratosis congenita family leads to cellular senescence and loss of CD34+ hematopoietic stem cells not reversible by mTOR-inhibition
Clemens Stockklausner, Simon Raffel, Julia Klermund, et al.
Journal of Inherited Metabolic Disease
|
November 18, 2016
Proposed recommendations for diagnosing and managing individuals with glutaric aciduria type I: second revision
Nikolas Boy, Chris Mühlhausen, Esther M Maier, et al.
European Journal of Human Genetics : EJHG
|
January 19, 2018
Compound heterozygous SPATA5 variants in four families and functional studies of SPATA5 deficiency
Sanna Puusepp, Reka Kovacs-Nagy, Bader Alhaddad, et al.
Journal of Inherited Metabolic Disease
|
June 8, 2026
Prevalence, Disease Onset and Clinical Outcome in Arginase 1 Deficiency: Cross-Border Surveillance in Germany, Austria, and Switzerland
Svenja Scharre, Annette L Hess, Florian Gleich, et al.
Page
of 39