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Neuropediatrics
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November 24, 2020
Thiamine Pyrophosphokinase Deficiency due to Mutations in the TPK1 Gene: A Rare, Treatable Neurodegenerative Disorder
Christina T Rüsch, Saskia B Wortmann, Reka Kovacs-Nagy, et al.
Stem Cell Research
|
January 7, 2026
Generation of two human iPSC lines from fibroblasts of BPAN patients carrying pathogenic variants in the WDR45 gene
Gemma Gasparini, Carolin Kraus, Ejona Rusha, et al.
The Laryngoscope
|
February 21, 2022
Mitochondrial Disease and Hearing Loss in Children: A Systematic Review
Sebastian Roesch, Anna O'Sullivan, Georg Zimmermann, et al.
Neuropediatrics
|
January 14, 2021
Cytosolic Phosphoenolpyruvate Carboxykinase Deficiency: Cause of Hypoglycemia-Induced Seizure and Death
Johanna Becker, Nikolaus A Haas, Stefan Vlaho, et al.
JIMD Reports
|
May 8, 2023
Patient-reported outcomes on empagliflozin treatment in glycogen storage disease type Ib: An international questionnaire study
Sarah C Grünert, Annieke Venema, Jamas LaFreniere, et al.
Journal of Inherited Metabolic Disease
|
September 10, 2005
Biochemical characteristics and increased tetraglucoside excretion in patients with phosphorylase kinase deficiency
E Morava, S B Wortmann, H Zweers van Essen, et al.
Neuropediatrics
|
October 6, 2025
Exploring Secondary Biotinidase Deficiency and Biotin Supplementation in PMM2-CDG
Nastassja Himmelreich, Sven F Garbade, Jürgen G Okun, et al.
Applied Radiation and Isotopes : Including Data, Instrumentation and Methods for Use in Agriculture, Industry and Medicine
|
August 30, 2011
Dosimetric feasibility study for an extracorporeal BNCT application on liver metastases at the TRIGA Mainz
M Blaickner, J V Kratz, S Minouchehr, et al.
Journal of Inherited Metabolic Disease
|
January 9, 2013
Inborn errors of metabolism with 3-methylglutaconic aciduria as discriminative feature: proper classification and nomenclature
Saskia B Wortmann, Marinus Duran, Yair Anikster, et al.
Orphanet Journal of Rare Diseases
|
July 22, 2018
Fatal pitfalls in newborn screening for mitochondrial trifunctional protein (MTP)/long-chain 3-Hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency
Amelie S Lotz-Havla, Wulf Röschinger, Katharina Schiergens, et al.
Page
of 21
Search research articles
Search
Showing results (41-50 of 205) with videos related to
Sort By:
Page
of 21
Neuropediatrics
|
November 24, 2020
Thiamine Pyrophosphokinase Deficiency due to Mutations in the TPK1 Gene: A Rare, Treatable Neurodegenerative Disorder
Christina T Rüsch, Saskia B Wortmann, Reka Kovacs-Nagy, et al.
Stem Cell Research
|
January 7, 2026
Generation of two human iPSC lines from fibroblasts of BPAN patients carrying pathogenic variants in the WDR45 gene
Gemma Gasparini, Carolin Kraus, Ejona Rusha, et al.
The Laryngoscope
|
February 21, 2022
Mitochondrial Disease and Hearing Loss in Children: A Systematic Review
Sebastian Roesch, Anna O'Sullivan, Georg Zimmermann, et al.
Neuropediatrics
|
January 14, 2021
Cytosolic Phosphoenolpyruvate Carboxykinase Deficiency: Cause of Hypoglycemia-Induced Seizure and Death
Johanna Becker, Nikolaus A Haas, Stefan Vlaho, et al.
JIMD Reports
|
May 8, 2023
Patient-reported outcomes on empagliflozin treatment in glycogen storage disease type Ib: An international questionnaire study
Sarah C Grünert, Annieke Venema, Jamas LaFreniere, et al.
Journal of Inherited Metabolic Disease
|
September 10, 2005
Biochemical characteristics and increased tetraglucoside excretion in patients with phosphorylase kinase deficiency
E Morava, S B Wortmann, H Zweers van Essen, et al.
Neuropediatrics
|
October 6, 2025
Exploring Secondary Biotinidase Deficiency and Biotin Supplementation in PMM2-CDG
Nastassja Himmelreich, Sven F Garbade, Jürgen G Okun, et al.
Applied Radiation and Isotopes : Including Data, Instrumentation and Methods for Use in Agriculture, Industry and Medicine
|
August 30, 2011
Dosimetric feasibility study for an extracorporeal BNCT application on liver metastases at the TRIGA Mainz
M Blaickner, J V Kratz, S Minouchehr, et al.
Journal of Inherited Metabolic Disease
|
January 9, 2013
Inborn errors of metabolism with 3-methylglutaconic aciduria as discriminative feature: proper classification and nomenclature
Saskia B Wortmann, Marinus Duran, Yair Anikster, et al.
Orphanet Journal of Rare Diseases
|
July 22, 2018
Fatal pitfalls in newborn screening for mitochondrial trifunctional protein (MTP)/long-chain 3-Hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency
Amelie S Lotz-Havla, Wulf Röschinger, Katharina Schiergens, et al.
Page
of 21