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Molecular Genetics and Metabolism
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February 28, 2026
Management of pegvaliase-related skin concerns: best practice recommendations using a modified Delphi approach
Alvaro Hermida Ameijeiras, Erika Vucko, Cary O Harding, et al.
Orphanet Journal of Rare Diseases
|
July 6, 2018
Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study
Nicola Longo, Roberto Zori, Melissa P Wasserstein, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 18, 2025
Safety and efficacy of pegtibatinase enzyme replacement therapy in adults with classical homocystinuria in the COMPOSE® phase 1/2 randomized trial
Can Ficicioglu, Janet A Thomas, Jaya Ganesh, et al.
European Journal of Pediatrics
|
September 25, 2007
Infantile cardiomyopathy caused by the T14709C mutation in the mitochondrial tRNA glutamic acid gene
Johan L K Van Hove, Cynthia Freehauf, Shelley Miyamoto, et al.
Molecular Genetics and Metabolism
|
May 23, 2008
The mitochondrial 13513G>A mutation is associated with Leigh disease phenotypes independent of complex I deficiency in muscle
Ariel Brautbar, Jing Wang, Jose E Abdenur, et al.
Molecular Genetics and Metabolism
|
December 27, 2024
Feasibility of newborn screening for pyridoxine-dependent epilepsy
Kristine Pauly, Michael Woontner, Jose E Abdenur, et al.
The Journal of Pediatrics
|
December 13, 2016
Mucopolysaccharidosis Type I Newborn Screening: Best Practices for Diagnosis and Management
Lorne A Clarke, Andrea M Atherton, Barbara K Burton, et al.
Molecular Genetics and Metabolism
|
May 24, 2011
Constitutive induction of pro-inflammatory and chemotactic cytokines in cystathionine beta-synthase deficient homocystinuria
Amy K Keating, Cynthia Freehauf, Hua Jiang, et al.
Molecular Genetics and Metabolism Reports
|
May 2, 2024
Pegvaliase for the treatment of phenylketonuria: Final results of a long-term phase 3 clinical trial program
Cary O Harding, Nicola Longo, Hope Northrup, et al.
Pediatric Research
|
May 11, 2010
Succinyl-CoA ligase deficiency: a mitochondrial hepatoencephalomyopathy
Johan L K Van Hove, Margarita S Saenz, Janet A Thomas, et al.
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Showing results (11-20 of 26) with videos related to
Sort By:
Page
of 3
Molecular Genetics and Metabolism
|
February 28, 2026
Management of pegvaliase-related skin concerns: best practice recommendations using a modified Delphi approach
Alvaro Hermida Ameijeiras, Erika Vucko, Cary O Harding, et al.
Orphanet Journal of Rare Diseases
|
July 6, 2018
Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study
Nicola Longo, Roberto Zori, Melissa P Wasserstein, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 18, 2025
Safety and efficacy of pegtibatinase enzyme replacement therapy in adults with classical homocystinuria in the COMPOSE® phase 1/2 randomized trial
Can Ficicioglu, Janet A Thomas, Jaya Ganesh, et al.
European Journal of Pediatrics
|
September 25, 2007
Infantile cardiomyopathy caused by the T14709C mutation in the mitochondrial tRNA glutamic acid gene
Johan L K Van Hove, Cynthia Freehauf, Shelley Miyamoto, et al.
Molecular Genetics and Metabolism
|
May 23, 2008
The mitochondrial 13513G>A mutation is associated with Leigh disease phenotypes independent of complex I deficiency in muscle
Ariel Brautbar, Jing Wang, Jose E Abdenur, et al.
Molecular Genetics and Metabolism
|
December 27, 2024
Feasibility of newborn screening for pyridoxine-dependent epilepsy
Kristine Pauly, Michael Woontner, Jose E Abdenur, et al.
The Journal of Pediatrics
|
December 13, 2016
Mucopolysaccharidosis Type I Newborn Screening: Best Practices for Diagnosis and Management
Lorne A Clarke, Andrea M Atherton, Barbara K Burton, et al.
Molecular Genetics and Metabolism
|
May 24, 2011
Constitutive induction of pro-inflammatory and chemotactic cytokines in cystathionine beta-synthase deficient homocystinuria
Amy K Keating, Cynthia Freehauf, Hua Jiang, et al.
Molecular Genetics and Metabolism Reports
|
May 2, 2024
Pegvaliase for the treatment of phenylketonuria: Final results of a long-term phase 3 clinical trial program
Cary O Harding, Nicola Longo, Hope Northrup, et al.
Pediatric Research
|
May 11, 2010
Succinyl-CoA ligase deficiency: a mitochondrial hepatoencephalomyopathy
Johan L K Van Hove, Margarita S Saenz, Janet A Thomas, et al.
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of 3