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Journal of Nephrology
|
December 19, 2024
Epidemiology and early predictors of Fabry nephropathy: evaluation of long-term outcomes from a national Fabry centre
Fahmida Mannan, Rajkumar Chinnadurai, Ryan Wiltshire, et al.
Orphanet Journal of Rare Diseases
|
September 4, 2023
Clinical, biochemical and molecular analysis in a cohort of individuals with gyrate atrophy
Eleanor Palmer, Karolina M Stepien, Christopher Campbell, et al.
Nutrients
|
April 30, 2021
Growth and Body Composition in PKU Children-A Three-Year Prospective Study Comparing the Effects of L-Amino Acid to Glycomacropeptide Protein Substitutes
Anne Daly, Wolfgang Högler, Nicola Crabtree, et al.
Molecular Genetics and Metabolism Reports
|
November 24, 2021
A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trials
Barbara K Burton, Anne Skalicky, Christoph Baerwald, et al.
Nutrients
|
July 2, 2021
A Three-Year Longitudinal Study Comparing Bone Mass, Density, and Geometry Measured by DXA, pQCT, and Bone Turnover Markers in Children with PKU Taking L-Amino Acid or Glycomacropeptide Protein Substitutes
Anne Daly, Wolfgang Högler, Nicola Crabtree, et al.
Investigative Ophthalmology & Visual Science
|
January 28, 2017
Novel PEX11B Mutations Extend the Peroxisome Biogenesis Disorder 14B Phenotypic Spectrum and Underscore Congenital Cataract as an Early Feature
Rachel L Taylor, Mark T Handley, Sarah Waller, et al.
Nutrients
|
November 3, 2020
Dietary Management, Clinical Status and Outcome of Patients with Citrin Deficiency in the UK
Alex Pinto, Catherine Ashmore, Spyros Batzios, et al.
Molecular Genetics and Metabolism
|
May 20, 2019
International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria
Ania C Muntau, Darius J Adams, Amaya Bélanger-Quintana, et al.
Archives of Disease in Childhood
|
May 5, 2017
Diagnosing childhood-onset inborn errors of metabolism by next-generation sequencing
Arunabha Ghosh, Helene Schlecht, Lesley E Heptinstall, et al.
Orphanet Journal of Rare Diseases
|
December 6, 2019
Emotional and behavioral problems, quality of life and metabolic control in NTBC-treated Tyrosinemia type 1 patients
Kimber van Vliet, Willem G van Ginkel, Rianne Jahja, et al.
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Search research articles
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Showing results (11-20 of 23) with videos related to
Sort By:
Page
of 3
Journal of Nephrology
|
December 19, 2024
Epidemiology and early predictors of Fabry nephropathy: evaluation of long-term outcomes from a national Fabry centre
Fahmida Mannan, Rajkumar Chinnadurai, Ryan Wiltshire, et al.
Orphanet Journal of Rare Diseases
|
September 4, 2023
Clinical, biochemical and molecular analysis in a cohort of individuals with gyrate atrophy
Eleanor Palmer, Karolina M Stepien, Christopher Campbell, et al.
Nutrients
|
April 30, 2021
Growth and Body Composition in PKU Children-A Three-Year Prospective Study Comparing the Effects of L-Amino Acid to Glycomacropeptide Protein Substitutes
Anne Daly, Wolfgang Högler, Nicola Crabtree, et al.
Molecular Genetics and Metabolism Reports
|
November 24, 2021
A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trials
Barbara K Burton, Anne Skalicky, Christoph Baerwald, et al.
Nutrients
|
July 2, 2021
A Three-Year Longitudinal Study Comparing Bone Mass, Density, and Geometry Measured by DXA, pQCT, and Bone Turnover Markers in Children with PKU Taking L-Amino Acid or Glycomacropeptide Protein Substitutes
Anne Daly, Wolfgang Högler, Nicola Crabtree, et al.
Investigative Ophthalmology & Visual Science
|
January 28, 2017
Novel PEX11B Mutations Extend the Peroxisome Biogenesis Disorder 14B Phenotypic Spectrum and Underscore Congenital Cataract as an Early Feature
Rachel L Taylor, Mark T Handley, Sarah Waller, et al.
Nutrients
|
November 3, 2020
Dietary Management, Clinical Status and Outcome of Patients with Citrin Deficiency in the UK
Alex Pinto, Catherine Ashmore, Spyros Batzios, et al.
Molecular Genetics and Metabolism
|
May 20, 2019
International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria
Ania C Muntau, Darius J Adams, Amaya Bélanger-Quintana, et al.
Archives of Disease in Childhood
|
May 5, 2017
Diagnosing childhood-onset inborn errors of metabolism by next-generation sequencing
Arunabha Ghosh, Helene Schlecht, Lesley E Heptinstall, et al.
Orphanet Journal of Rare Diseases
|
December 6, 2019
Emotional and behavioral problems, quality of life and metabolic control in NTBC-treated Tyrosinemia type 1 patients
Kimber van Vliet, Willem G van Ginkel, Rianne Jahja, et al.
Page
of 3