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Plos One
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June 28, 2024
Quality considerations and major pitfalls for high throughput DNA-based newborn screening for severe combined immunodeficiency and spinal muscular atrophy
Jessica Bzdok, Ludwig Czibere, Siegfried Burggraf, et al.
American Journal of Medical Genetics. Part A
|
February 22, 2005
Genotypic and phenotypic characterization of Noonan syndrome: new data and review of the literature
Marjolijn Jongmans, Erik A Sistermans, Alwin Rikken, et al.
Molecular Genetics and Metabolism
|
May 18, 2005
Reduced incidence of severe metabolic crisis or death in children with medium chain acyl-CoA dehydrogenase deficiency homozygous for c.985A>G identified by neonatal screening
Uta Nennstiel-Ratzel, Stephan Arenz, Esther M Maier, et al.
Neuropediatrics
|
January 8, 2025
Intravitreal Enzyme Replacement Therapy Slows Retinopathy in Late Infantile Ceroid Lipofuscinosis Type 2
Claudia S Priglinger, Carolina Courage, Amelie S Lotz-Havla, et al.
Clinical Genetics
|
February 25, 2023
Free carnitine concentrations and biochemical parameters in medium-chain acyl-CoA dehydrogenase deficiency: Genotype-phenotype correlation
Katharina J Weiss, Ursula Berger, Maliha Haider, et al.
Molecular Genetics and Metabolism Reports
|
June 28, 2021
Newborn screening for carnitine transporter defect in Bavaria and the long-term follow-up of the identified newborns and mothers: Assessing the benefit and possible harm based on 19 ½ years of experience
Katharina A Schiergens, Katharina J Weiss, Wulf Röschinger, et al.
Human Molecular Genetics
|
February 20, 2009
Protein misfolding is the molecular mechanism underlying MCADD identified in newborn screening
Esther M Maier, Søren W Gersting, Kristina F Kemter, et al.
Journal of Inherited Metabolic Disease
|
September 14, 2012
Low lysine diet in glutaric aciduria type I--effect on anthropometric and biochemical follow-up parameters
Nikolas Boy, Gisela Haege, Jana Heringer, et al.
Journal of Neurology
|
March 31, 2020
Retinal axonal degeneration in Niemann-Pick type C disease
Joachim Havla, Marlene Moser, Clara Sztatecsny, et al.
American Journal of Human Genetics
|
November 24, 2001
Mutations in the proenteropeptidase gene are the molecular cause of congenital enteropeptidase deficiency
Andreas Holzinger, Esther M Maier, Cornelius Bück, et al.
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of 7
Search research articles
Search
Showing results (21-30 of 64) with videos related to
Sort By:
Page
of 7
Plos One
|
June 28, 2024
Quality considerations and major pitfalls for high throughput DNA-based newborn screening for severe combined immunodeficiency and spinal muscular atrophy
Jessica Bzdok, Ludwig Czibere, Siegfried Burggraf, et al.
American Journal of Medical Genetics. Part A
|
February 22, 2005
Genotypic and phenotypic characterization of Noonan syndrome: new data and review of the literature
Marjolijn Jongmans, Erik A Sistermans, Alwin Rikken, et al.
Molecular Genetics and Metabolism
|
May 18, 2005
Reduced incidence of severe metabolic crisis or death in children with medium chain acyl-CoA dehydrogenase deficiency homozygous for c.985A>G identified by neonatal screening
Uta Nennstiel-Ratzel, Stephan Arenz, Esther M Maier, et al.
Neuropediatrics
|
January 8, 2025
Intravitreal Enzyme Replacement Therapy Slows Retinopathy in Late Infantile Ceroid Lipofuscinosis Type 2
Claudia S Priglinger, Carolina Courage, Amelie S Lotz-Havla, et al.
Clinical Genetics
|
February 25, 2023
Free carnitine concentrations and biochemical parameters in medium-chain acyl-CoA dehydrogenase deficiency: Genotype-phenotype correlation
Katharina J Weiss, Ursula Berger, Maliha Haider, et al.
Molecular Genetics and Metabolism Reports
|
June 28, 2021
Newborn screening for carnitine transporter defect in Bavaria and the long-term follow-up of the identified newborns and mothers: Assessing the benefit and possible harm based on 19 ½ years of experience
Katharina A Schiergens, Katharina J Weiss, Wulf Röschinger, et al.
Human Molecular Genetics
|
February 20, 2009
Protein misfolding is the molecular mechanism underlying MCADD identified in newborn screening
Esther M Maier, Søren W Gersting, Kristina F Kemter, et al.
Journal of Inherited Metabolic Disease
|
September 14, 2012
Low lysine diet in glutaric aciduria type I--effect on anthropometric and biochemical follow-up parameters
Nikolas Boy, Gisela Haege, Jana Heringer, et al.
Journal of Neurology
|
March 31, 2020
Retinal axonal degeneration in Niemann-Pick type C disease
Joachim Havla, Marlene Moser, Clara Sztatecsny, et al.
American Journal of Human Genetics
|
November 24, 2001
Mutations in the proenteropeptidase gene are the molecular cause of congenital enteropeptidase deficiency
Andreas Holzinger, Esther M Maier, Cornelius Bück, et al.
Page
of 7