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Frontiers in Pediatrics
|
March 15, 2017
Humoral Immunodeficiency with Hypotonia, Feeding Difficulties, Enteropathy, and Mild Eczema Caused by a Classical <i>FOXP3</i> Mutation
Paul Tuijnenburg, Eloy Cuadrado, Annet M Bosch, et al.
Journal of Inherited Metabolic Disease
|
April 26, 2019
Limited data to evaluate real-world effectiveness of enzyme replacement therapy for mucopolysaccharidosis type I
Gé-Ann Kuiper, Stephanie C M Nijmeijer, Manouck J M Roelofs, et al.
Orphanet Journal of Rare Diseases
|
June 2, 2023
The challenges of classical galactosemia: HRQoL in pediatric and adult patients
Merel E Hermans, Hedy A van Oers, Gert J Geurtsen, et al.
Molecular Genetics and Metabolism
|
January 10, 2017
Nine years of newborn screening for classical galactosemia in the Netherlands: Effectiveness of screening methods, and identification of patients with previously unreported phenotypes
Lindsey Welling, Anita Boelen, Terry G J Derks, et al.
Reproductive Sciences (Thousand Oaks, Calif.)
|
December 20, 2007
Heterozygosity for the classical galactosemia mutation does not affect ovarian reserve and menopausal age
Erik A H Knauff, Renate Richardus, Marinus J Eijkemans, et al.
Molecular Genetics and Metabolism
|
March 28, 2018
Recommendations for newborn screening for galactokinase deficiency: A systematic review and evaluation of Dutch newborn screening data
Kevin Stroek, Marelle J Bouva, Peter C J I Schielen, et al.
Journal of Inherited Metabolic Disease
|
August 2, 2025
Effectiveness of Pyridoxal-5'-Phosphate in PNPO Deficiency: A Systematic Review
Nina N Stolwijk, Laura van Dussen, Niels D Reijnhout, et al.
Orphanet Journal of Rare Diseases
|
November 27, 2014
Rare inborn errors of metabolism with movement disorders: a case study to evaluate the impact upon quality of life and adaptive functioning
Hendriekje Eggink, Anouk Kuiper, Kathryn J Peall, et al.
Orphanet Journal of Rare Diseases
|
January 30, 2016
The neonatal tetrahydrobiopterin loading test in phenylketonuria: what is the predictive value?
Karen Anjema, Floris C Hofstede, Annet M Bosch, et al.
Orphanet Journal of Rare Diseases
|
February 28, 2013
Clinical pathways for inborn errors of metabolism: warranted and feasible
Serwet Demirdas, Imke N van Kessel, Marjolein J Korndewal, et al.
Page
of 11
Search research articles
Search
Showing results (31-40 of 103) with videos related to
Sort By:
Page
of 11
Frontiers in Pediatrics
|
March 15, 2017
Humoral Immunodeficiency with Hypotonia, Feeding Difficulties, Enteropathy, and Mild Eczema Caused by a Classical <i>FOXP3</i> Mutation
Paul Tuijnenburg, Eloy Cuadrado, Annet M Bosch, et al.
Journal of Inherited Metabolic Disease
|
April 26, 2019
Limited data to evaluate real-world effectiveness of enzyme replacement therapy for mucopolysaccharidosis type I
Gé-Ann Kuiper, Stephanie C M Nijmeijer, Manouck J M Roelofs, et al.
Orphanet Journal of Rare Diseases
|
June 2, 2023
The challenges of classical galactosemia: HRQoL in pediatric and adult patients
Merel E Hermans, Hedy A van Oers, Gert J Geurtsen, et al.
Molecular Genetics and Metabolism
|
January 10, 2017
Nine years of newborn screening for classical galactosemia in the Netherlands: Effectiveness of screening methods, and identification of patients with previously unreported phenotypes
Lindsey Welling, Anita Boelen, Terry G J Derks, et al.
Reproductive Sciences (Thousand Oaks, Calif.)
|
December 20, 2007
Heterozygosity for the classical galactosemia mutation does not affect ovarian reserve and menopausal age
Erik A H Knauff, Renate Richardus, Marinus J Eijkemans, et al.
Molecular Genetics and Metabolism
|
March 28, 2018
Recommendations for newborn screening for galactokinase deficiency: A systematic review and evaluation of Dutch newborn screening data
Kevin Stroek, Marelle J Bouva, Peter C J I Schielen, et al.
Journal of Inherited Metabolic Disease
|
August 2, 2025
Effectiveness of Pyridoxal-5'-Phosphate in PNPO Deficiency: A Systematic Review
Nina N Stolwijk, Laura van Dussen, Niels D Reijnhout, et al.
Orphanet Journal of Rare Diseases
|
November 27, 2014
Rare inborn errors of metabolism with movement disorders: a case study to evaluate the impact upon quality of life and adaptive functioning
Hendriekje Eggink, Anouk Kuiper, Kathryn J Peall, et al.
Orphanet Journal of Rare Diseases
|
January 30, 2016
The neonatal tetrahydrobiopterin loading test in phenylketonuria: what is the predictive value?
Karen Anjema, Floris C Hofstede, Annet M Bosch, et al.
Orphanet Journal of Rare Diseases
|
February 28, 2013
Clinical pathways for inborn errors of metabolism: warranted and feasible
Serwet Demirdas, Imke N van Kessel, Marjolein J Korndewal, et al.
Page
of 11