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Molecular Genetics and Metabolism
|
January 20, 2024
Impact of genetic and non-genetic factors on phenotypic diversity in NBAS-associated disease
Nicole Hammann, Dominic Lenz, Ivo Baric, et al.
Neurology
|
June 2, 2023
Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial
Amel Karaa, Enrico Bertini, Valerio Carelli, et al.
Molecular Genetics and Metabolism
|
September 26, 2016
Nutritional interventions in primary mitochondrial disorders: Developing an evidence base
Kathryn M Camp, Danuta Krotoski, Melissa A Parisi, et al.
Orphanet Journal of Rare Diseases
|
November 22, 2024
Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial
Amel Karaa, Enrico Bertini, Valerio Carelli, et al.
Brain : a Journal of Neurology
|
December 27, 2021
Bi-allelic loss-of-function OBSCN variants predispose individuals to severe recurrent rhabdomyolysis
Macarena Cabrera-Serrano, Laure Caccavelli, Marco Savarese, et al.
Nature Metabolism
|
November 23, 2023
Anomalous peroxidase activity of cytochrome c is the primary pathogenic target in Barth syndrome
Valerian E Kagan, Yulia Y Tyurina, Karolina Mikulska-Ruminska, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
January 15, 2026
Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: interim results from the Phase 3 APHENITY Extension Study
Francjan van Spronsen, Heidi Peters, Lali Margvelashvili, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
November 26, 2019
Defining clinical subgroups and genotype-phenotype correlations in NBAS-associated disease across 110 patients
Christian Staufner, Bianca Peters, Matias Wagner, et al.
American Journal of Human Genetics
|
July 16, 2020
The Genetic Landscape and Epidemiology of Phenylketonuria
Alicia Hillert, Yair Anikster, Amaya Belanger-Quintana, et al.
Molecular Genetics and Metabolism
|
July 3, 2024
Frontiers in congenital disorders of glycosylation consortium, a cross-sectional study report at year 5 of 280 individuals in the natural history cohort
Christina Lam, Fernando Scaglia, Gerard T Berry, et al.
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of 30
Search research articles
Search
Showing results (281-290 of 295) with videos related to
Sort By:
Page
of 30
Molecular Genetics and Metabolism
|
January 20, 2024
Impact of genetic and non-genetic factors on phenotypic diversity in NBAS-associated disease
Nicole Hammann, Dominic Lenz, Ivo Baric, et al.
Neurology
|
June 2, 2023
Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial
Amel Karaa, Enrico Bertini, Valerio Carelli, et al.
Molecular Genetics and Metabolism
|
September 26, 2016
Nutritional interventions in primary mitochondrial disorders: Developing an evidence base
Kathryn M Camp, Danuta Krotoski, Melissa A Parisi, et al.
Orphanet Journal of Rare Diseases
|
November 22, 2024
Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial
Amel Karaa, Enrico Bertini, Valerio Carelli, et al.
Brain : a Journal of Neurology
|
December 27, 2021
Bi-allelic loss-of-function OBSCN variants predispose individuals to severe recurrent rhabdomyolysis
Macarena Cabrera-Serrano, Laure Caccavelli, Marco Savarese, et al.
Nature Metabolism
|
November 23, 2023
Anomalous peroxidase activity of cytochrome c is the primary pathogenic target in Barth syndrome
Valerian E Kagan, Yulia Y Tyurina, Karolina Mikulska-Ruminska, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
January 15, 2026
Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: interim results from the Phase 3 APHENITY Extension Study
Francjan van Spronsen, Heidi Peters, Lali Margvelashvili, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
November 26, 2019
Defining clinical subgroups and genotype-phenotype correlations in NBAS-associated disease across 110 patients
Christian Staufner, Bianca Peters, Matias Wagner, et al.
American Journal of Human Genetics
|
July 16, 2020
The Genetic Landscape and Epidemiology of Phenylketonuria
Alicia Hillert, Yair Anikster, Amaya Belanger-Quintana, et al.
Molecular Genetics and Metabolism
|
July 3, 2024
Frontiers in congenital disorders of glycosylation consortium, a cross-sectional study report at year 5 of 280 individuals in the natural history cohort
Christina Lam, Fernando Scaglia, Gerard T Berry, et al.
Page
of 30