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JIMD Reports
|
May 12, 2021
Real-world management of maple syrup urine disease (MSUD) metabolic decompensations with branched chain amino acid-free formulas in France and Germany: A retrospective observational study
Pascale de Lonlay, Roland Posset, Ulrike Mütze, et al.
Molecular Genetics and Metabolism
|
February 28, 2022
What are the clues for an inherited metabolic disorder in Reye syndrome? A single Centre study of 58 children
Violette Goetz, David Dawei Yang, Florence Lacaille, et al.
Molecular Genetics and Metabolism Reports
|
November 17, 2022
Initial presentation, management and follow-up data of 33 treated patients with hereditary tyrosinemia type 1 in the absence of newborn screening
Hela Hajji, Apolline Imbard, Anne Spraul, et al.
Orphanet Journal of Rare Diseases
|
November 15, 2022
Efficacy and pharmacokinetics of betaine in CBS and cblC deficiencies: a cross-over randomized controlled trial
Apolline Imbard, Artemis Toumazi, Sophie Magréault, et al.
Frontiers in Pediatrics
|
March 22, 2021
West Syndrome Is an Exceptional Presentation of Pyridoxine- and Pyridoxal Phosphate-Dependent Epilepsy: Data From a French Cohort and Review of the Literature
Marc Gibaud, Magalie Barth, Jérémie Lefranc, et al.
Nature Medicine
|
May 29, 2023
Enasidenib treatment in two individuals with D-2-hydroxyglutaric aciduria carrying a germline IDH2 mutation
Birgit Geoerger, Manuel Schiff, Virginie Penard-Lacronique, et al.
European Journal of Medical Genetics
|
July 6, 2010
Further delineation of the 17p13.3 microdeletion involving YWHAE but distal to PAFAH1B1: four additional patients
Manuel Schiff, Andrée Delahaye, Joris Andrieux, et al.
Journal of Inherited Metabolic Disease
|
March 7, 2023
Successful treatment of severe MSUD in Bckdhb<sup>-/-</sup> mice with neonatal AAV gene therapy
Clément Pontoizeau, Clovis Gaborit, Nolan Tual, et al.
Molecular Genetics and Metabolism
|
February 14, 2018
Long-term liver disease in methylmalonic and propionic acidemias
Apolline Imbard, Nuria Garcia Segarra, Marine Tardieu, et al.
Molecular Genetics and Metabolism
|
July 16, 2023
Motor outcomes in patients with infantile and juvenile Pompe disease: Lessons from neurophysiological findings
Anaïs Brassier, Samia Pichard, Manuel Schiff, et al.
Page
of 16
Search research articles
Search
Showing results (71-80 of 159) with videos related to
Sort By:
Page
of 16
JIMD Reports
|
May 12, 2021
Real-world management of maple syrup urine disease (MSUD) metabolic decompensations with branched chain amino acid-free formulas in France and Germany: A retrospective observational study
Pascale de Lonlay, Roland Posset, Ulrike Mütze, et al.
Molecular Genetics and Metabolism
|
February 28, 2022
What are the clues for an inherited metabolic disorder in Reye syndrome? A single Centre study of 58 children
Violette Goetz, David Dawei Yang, Florence Lacaille, et al.
Molecular Genetics and Metabolism Reports
|
November 17, 2022
Initial presentation, management and follow-up data of 33 treated patients with hereditary tyrosinemia type 1 in the absence of newborn screening
Hela Hajji, Apolline Imbard, Anne Spraul, et al.
Orphanet Journal of Rare Diseases
|
November 15, 2022
Efficacy and pharmacokinetics of betaine in CBS and cblC deficiencies: a cross-over randomized controlled trial
Apolline Imbard, Artemis Toumazi, Sophie Magréault, et al.
Frontiers in Pediatrics
|
March 22, 2021
West Syndrome Is an Exceptional Presentation of Pyridoxine- and Pyridoxal Phosphate-Dependent Epilepsy: Data From a French Cohort and Review of the Literature
Marc Gibaud, Magalie Barth, Jérémie Lefranc, et al.
Nature Medicine
|
May 29, 2023
Enasidenib treatment in two individuals with D-2-hydroxyglutaric aciduria carrying a germline IDH2 mutation
Birgit Geoerger, Manuel Schiff, Virginie Penard-Lacronique, et al.
European Journal of Medical Genetics
|
July 6, 2010
Further delineation of the 17p13.3 microdeletion involving YWHAE but distal to PAFAH1B1: four additional patients
Manuel Schiff, Andrée Delahaye, Joris Andrieux, et al.
Journal of Inherited Metabolic Disease
|
March 7, 2023
Successful treatment of severe MSUD in Bckdhb<sup>-/-</sup> mice with neonatal AAV gene therapy
Clément Pontoizeau, Clovis Gaborit, Nolan Tual, et al.
Molecular Genetics and Metabolism
|
February 14, 2018
Long-term liver disease in methylmalonic and propionic acidemias
Apolline Imbard, Nuria Garcia Segarra, Marine Tardieu, et al.
Molecular Genetics and Metabolism
|
July 16, 2023
Motor outcomes in patients with infantile and juvenile Pompe disease: Lessons from neurophysiological findings
Anaïs Brassier, Samia Pichard, Manuel Schiff, et al.
Page
of 16