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Journal of Inherited Metabolic Disease
|
February 19, 2026
Maternal and Fetal Outcomes in Imiglucerase-Treated Patients With Gaucher Disease: Real-World Evidence From the International Collaborative Gaucher Group (ICGG) Gaucher Registry Pregnancy Sub-Registry
Shoshana Revel-Vilk, Patrick Deegan, Debra Day-Salvatore, et al.
Clinical Chemistry
|
February 17, 2019
Increased Clinical Sensitivity and Specificity of Plasma Protein <i>N</i>-Glycan Profiling for Diagnosing Congenital Disorders of Glycosylation by Use of Flow Injection-Electrospray Ionization-Quadrupole Time-of-Flight Mass Spectrometry
Jie Chen, Xueli Li, Andrew Edmondson, et al.
Biochimica Et Biophysica Acta
|
February 8, 2011
Clinical and molecular characterization of five patients with succinyl-CoA:3-ketoacid CoA transferase (SCOT) deficiency
Toshiyuki Fukao, Jörn Oliver Sass, Petri Kursula, et al.
The Journal of Pediatrics
|
December 24, 2018
Early Indicators of Creatine Transporter Deficiency
Judith S Miller, Rebecca P Thomas, Amanda Bennett, et al.
International Journal of Neonatal Screening
|
August 25, 2020
The Importance of Succinylacetone: Tyrosinemia Type I Presenting with Hyperinsulinism and Multiorgan Failure Following Normal Newborn Screening
Jessica R C Priestley, Hana Alharbi, Katharine Press Callahan, et al.
International Journal of Neonatal Screening
|
March 26, 2025
Five-Year Outcomes of Patients with Pompe Disease Identified by the Pennsylvania Newborn Screen
Hayley A Ron, Owen Kane, Rose Guo, 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.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
January 26, 2021
Transforming the clinical outcome in CRIM-negative infantile Pompe disease identified via newborn screening: the benefits of early treatment with enzyme replacement therapy and immune tolerance induction
Cindy Li, Ankit K Desai, Punita Gupta, 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.
Orphanet Journal of Rare Diseases
|
February 3, 2018
Consensus guidelines for newborn screening, diagnosis and treatment of infantile Krabbe disease
Jennifer M Kwon, Dietrich Matern, Joanne Kurtzberg, et al.
Page
of 11
Search research articles
Search
Showing results (61-70 of 110) with videos related to
Sort By:
Page
of 11
Journal of Inherited Metabolic Disease
|
February 19, 2026
Maternal and Fetal Outcomes in Imiglucerase-Treated Patients With Gaucher Disease: Real-World Evidence From the International Collaborative Gaucher Group (ICGG) Gaucher Registry Pregnancy Sub-Registry
Shoshana Revel-Vilk, Patrick Deegan, Debra Day-Salvatore, et al.
Clinical Chemistry
|
February 17, 2019
Increased Clinical Sensitivity and Specificity of Plasma Protein <i>N</i>-Glycan Profiling for Diagnosing Congenital Disorders of Glycosylation by Use of Flow Injection-Electrospray Ionization-Quadrupole Time-of-Flight Mass Spectrometry
Jie Chen, Xueli Li, Andrew Edmondson, et al.
Biochimica Et Biophysica Acta
|
February 8, 2011
Clinical and molecular characterization of five patients with succinyl-CoA:3-ketoacid CoA transferase (SCOT) deficiency
Toshiyuki Fukao, Jörn Oliver Sass, Petri Kursula, et al.
The Journal of Pediatrics
|
December 24, 2018
Early Indicators of Creatine Transporter Deficiency
Judith S Miller, Rebecca P Thomas, Amanda Bennett, et al.
International Journal of Neonatal Screening
|
August 25, 2020
The Importance of Succinylacetone: Tyrosinemia Type I Presenting with Hyperinsulinism and Multiorgan Failure Following Normal Newborn Screening
Jessica R C Priestley, Hana Alharbi, Katharine Press Callahan, et al.
International Journal of Neonatal Screening
|
March 26, 2025
Five-Year Outcomes of Patients with Pompe Disease Identified by the Pennsylvania Newborn Screen
Hayley A Ron, Owen Kane, Rose Guo, 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.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
January 26, 2021
Transforming the clinical outcome in CRIM-negative infantile Pompe disease identified via newborn screening: the benefits of early treatment with enzyme replacement therapy and immune tolerance induction
Cindy Li, Ankit K Desai, Punita Gupta, 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.
Orphanet Journal of Rare Diseases
|
February 3, 2018
Consensus guidelines for newborn screening, diagnosis and treatment of infantile Krabbe disease
Jennifer M Kwon, Dietrich Matern, Joanne Kurtzberg, et al.
Page
of 11