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Human Molecular Genetics
|
March 5, 2002
A frequent mild mutation in ALG6 may exacerbate the clinical severity of patients with congenital disorder of glycosylation Ia (CDG-Ia) caused by phosphomannomutase deficiency
Vibeke Westphal, Susanne Kjaergaard, Els Schollen, et al.
Molecular Genetics and Metabolism Reports
|
June 30, 2026
Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy
Eugen Mengel, Laila Arash-Kaps, Stephanie Grunewald, et al.
Neuromuscular Disorders : NMD
|
January 17, 2021
Persistently elevated CK and lysosomal storage myopathy associated with mucolipin 1 defects
Alberto A Zambon, Alexandra Lemaigre, Rahul Phadke, et al.
JIMD Reports
|
March 14, 2022
Direct replacement of oral sodium benzoate with glycerol phenylbutyrate in children with urea cycle disorders
Mildrid Yeo, Preeya Rehsi, Megan Dorman, et al.
JIMD Reports
|
September 13, 2023
Clinical experience with glycerol phenylbutyrate in 20 patients with urea cycle disorders at a UK paediatric centre
Mildrid Yeo, Preeya Rehsi, Megan Dorman, et al.
Pediatric Neurology
|
September 13, 2011
Clinical neuroimaging features and outcome in molybdenum cofactor deficiency
Kayal Vijayakumar, Rox Gunny, Stephanie Grunewald, et al.
Journal of Proteome Research
|
June 8, 2013
A new method for the rapid diagnosis of protein N-linked congenital disorders of glycosylation
Wendy E Heywood, Philippa Mills, Stephanie Grunewald, et al.
JIMD Reports
|
July 5, 2023
Prolonged respiratory failure responds to conventional therapy in isolated homocysteine remethylation defects
Abigail Whitehouse, Preeya Rehsi, Louise Hartley, et al.
Journal of Inherited Metabolic Disease
|
November 17, 2011
Further delineation of pontocerebellar hypoplasia type 6 due to mutations in the gene encoding mitochondrial arginyl-tRNA synthetase, RARS2
Emma Glamuzina, Ruth Brown, Kieran Hogarth, et al.
Pediatric Nephrology (Berlin, Germany)
|
February 25, 2023
Transplantation in paediatric patients with MMA requires multidisciplinary approach for achievement of good clinical outcomes
Alicia Paessler, Miriam Cortes-Cerisuelo, Wayel Jassem, et al.
Page
of 6
Search research articles
Search
Showing results (11-20 of 59) with videos related to
Sort By:
Page
of 6
Human Molecular Genetics
|
March 5, 2002
A frequent mild mutation in ALG6 may exacerbate the clinical severity of patients with congenital disorder of glycosylation Ia (CDG-Ia) caused by phosphomannomutase deficiency
Vibeke Westphal, Susanne Kjaergaard, Els Schollen, et al.
Molecular Genetics and Metabolism Reports
|
June 30, 2026
Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy
Eugen Mengel, Laila Arash-Kaps, Stephanie Grunewald, et al.
Neuromuscular Disorders : NMD
|
January 17, 2021
Persistently elevated CK and lysosomal storage myopathy associated with mucolipin 1 defects
Alberto A Zambon, Alexandra Lemaigre, Rahul Phadke, et al.
JIMD Reports
|
March 14, 2022
Direct replacement of oral sodium benzoate with glycerol phenylbutyrate in children with urea cycle disorders
Mildrid Yeo, Preeya Rehsi, Megan Dorman, et al.
JIMD Reports
|
September 13, 2023
Clinical experience with glycerol phenylbutyrate in 20 patients with urea cycle disorders at a UK paediatric centre
Mildrid Yeo, Preeya Rehsi, Megan Dorman, et al.
Pediatric Neurology
|
September 13, 2011
Clinical neuroimaging features and outcome in molybdenum cofactor deficiency
Kayal Vijayakumar, Rox Gunny, Stephanie Grunewald, et al.
Journal of Proteome Research
|
June 8, 2013
A new method for the rapid diagnosis of protein N-linked congenital disorders of glycosylation
Wendy E Heywood, Philippa Mills, Stephanie Grunewald, et al.
JIMD Reports
|
July 5, 2023
Prolonged respiratory failure responds to conventional therapy in isolated homocysteine remethylation defects
Abigail Whitehouse, Preeya Rehsi, Louise Hartley, et al.
Journal of Inherited Metabolic Disease
|
November 17, 2011
Further delineation of pontocerebellar hypoplasia type 6 due to mutations in the gene encoding mitochondrial arginyl-tRNA synthetase, RARS2
Emma Glamuzina, Ruth Brown, Kieran Hogarth, et al.
Pediatric Nephrology (Berlin, Germany)
|
February 25, 2023
Transplantation in paediatric patients with MMA requires multidisciplinary approach for achievement of good clinical outcomes
Alicia Paessler, Miriam Cortes-Cerisuelo, Wayel Jassem, et al.
Page
of 6