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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.
Neuropediatrics
|
June 22, 2019
EPG5 Variants with Modest Functional Impact Result in an Ameliorated and Primarily Neurological Phenotype in a 3.5-Year-Old Patient with Vici Syndrome
Megan S Kane, Jia Zhao, Julie Muskett, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
January 4, 2014
Phenylalanine hydroxylase deficiency: diagnosis and management guideline
Jerry Vockley, Hans C Andersson, Kevin M Antshel, et al.
Molecular Genetics and Metabolism
|
September 17, 2017
Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2months to 2years
Susan A Berry, Nicola Longo, George A Diaz, et al.
Molecular Genetics and Metabolism
|
June 18, 2020
Laboratory monitoring of patients with hereditary tyrosinemia type I
Matthew J Schultz, Brian C Netzel, Rani H Singh, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
January 9, 2026
Phenylalanine hydroxylase deficiency diagnosis and management: A 2023 evidence-based clinical guideline of the American College of Medical Genetics and Genomics (ACMG)
Wendy E Smith, Susan A Berry, Kaitlyn Bloom, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
December 4, 2024
Phenylalanine hydroxylase deficiency diagnosis and management: A 2023 evidence-based clinical guideline of the American College of Medical Genetics and Genomics (ACMG)
Wendy E Smith, Susan A Berry, Kaitlyn Bloom, et al.
Molecular Genetics and Metabolism
|
September 16, 2018
Pharmacokinetics of glycerol phenylbutyrate in pediatric patients 2 months to 2 years of age with urea cycle disorders
Susan A Berry, Jerry Vockley, Alexander A Vinks, et al.
Pediatric Research
|
May 9, 2003
Rare disorders of metabolism with elevated butyryl- and isobutyryl-carnitine detected by tandem mass spectrometry newborn screening
Dwight D Koeberl, Sarah P Young, Niels S Gregersen, et al.
American Journal of Medical Genetics. Part A
|
May 7, 2011
Distinctive phenotype in 9 patients with deletion of chromosome 1q24-q25
Deepika D'Cunha Burkardt, Jill A Rosenfeld, Maria L Helgeson, et al.
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Search research articles
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Showing results (11-20 of 32) with videos related to
Sort By:
Page
of 4
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.
Neuropediatrics
|
June 22, 2019
EPG5 Variants with Modest Functional Impact Result in an Ameliorated and Primarily Neurological Phenotype in a 3.5-Year-Old Patient with Vici Syndrome
Megan S Kane, Jia Zhao, Julie Muskett, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
January 4, 2014
Phenylalanine hydroxylase deficiency: diagnosis and management guideline
Jerry Vockley, Hans C Andersson, Kevin M Antshel, et al.
Molecular Genetics and Metabolism
|
September 17, 2017
Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2months to 2years
Susan A Berry, Nicola Longo, George A Diaz, et al.
Molecular Genetics and Metabolism
|
June 18, 2020
Laboratory monitoring of patients with hereditary tyrosinemia type I
Matthew J Schultz, Brian C Netzel, Rani H Singh, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
January 9, 2026
Phenylalanine hydroxylase deficiency diagnosis and management: A 2023 evidence-based clinical guideline of the American College of Medical Genetics and Genomics (ACMG)
Wendy E Smith, Susan A Berry, Kaitlyn Bloom, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
December 4, 2024
Phenylalanine hydroxylase deficiency diagnosis and management: A 2023 evidence-based clinical guideline of the American College of Medical Genetics and Genomics (ACMG)
Wendy E Smith, Susan A Berry, Kaitlyn Bloom, et al.
Molecular Genetics and Metabolism
|
September 16, 2018
Pharmacokinetics of glycerol phenylbutyrate in pediatric patients 2 months to 2 years of age with urea cycle disorders
Susan A Berry, Jerry Vockley, Alexander A Vinks, et al.
Pediatric Research
|
May 9, 2003
Rare disorders of metabolism with elevated butyryl- and isobutyryl-carnitine detected by tandem mass spectrometry newborn screening
Dwight D Koeberl, Sarah P Young, Niels S Gregersen, et al.
American Journal of Medical Genetics. Part A
|
May 7, 2011
Distinctive phenotype in 9 patients with deletion of chromosome 1q24-q25
Deepika D'Cunha Burkardt, Jill A Rosenfeld, Maria L Helgeson, et al.
Page
of 4