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European Journal of Medical Genetics
|
June 30, 2023
A novel SLC5A6 homozygous variant in a family with multivitamin-dependent neurometabolic disorder: Phenotype expansion and long-term follow-up
Martino Montomoli, Annalisa Vetro, Flavia Tubili, et al.
JAMA Neurology
|
July 10, 2013
Mitochondrial encephalomyopathy due to a novel mutation in ACAD9
Caterina Garone, Maria Alice Donati, Michele Sacchini, et al.
Metabolic Brain Disease
|
June 4, 2017
The treatment of juvenile/adult GM1-gangliosidosis with Miglustat may reverse disease progression
Federica Deodato, Elena Procopio, Angelica Rampazzo, et al.
Molecular Genetics and Metabolism
|
July 5, 2017
A rare case of sterol-C4-methyl oxidase deficiency in a young Italian male: Biochemical and molecular characterization
Giulia Frisso, Monica Gelzo, Elena Procopio, et al.
International Journal of Cardiology
|
November 29, 2018
Clinical profile and outcome of cardiac involvement in MELAS syndrome
Alice Brambilla, Silvia Favilli, Iacopo Olivotto, et al.
BMC Medical Genomics
|
June 13, 2021
Bi-allelic variants in MTMR5/SBF1 cause Charcot-Marie-Tooth type 4B3 featuring mitochondrial dysfunction
Beatrice Berti, Giovanna Longo, Francesco Mari, et al.
Clinica Chimica Acta; International Journal of Clinical Chemistry
|
December 30, 2008
Rapid diagnostic testing procedures for lysosomal storage disorders: alpha-glucosidase and beta-galactosidase assays on dried blood spots
Nicoletta Gasparotto, Rosella Tomanin, Anna Chiara Frigo, et al.
Frontiers in Physiology
|
June 2, 2020
Advances in Stem Cell Modeling of Dystrophin-Associated Disease: Implications for the Wider World of Dilated Cardiomyopathy
Josè Manuel Pioner, Alessandra Fornaro, Raffaele Coppini, et al.
Metabolic Brain Disease
|
July 1, 2017
SSADH deficiency in an Italian family: a novel ALDH5A1 gene mutation affecting the succinic semialdehyde substrate binding site
Sara Leo, Concetta Capo, Bianca Maria Ciminelli, et al.
Molecular Genetics and Metabolism
|
December 3, 2019
Novel compound heterozygous pathogenic variants in nucleotide-binding protein like protein (NUBPL) cause leukoencephalopathy with multi-systemic involvement
Margherita Protasoni, Claudio Bruno, Maria Alice Donati, et al.
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of 9
Search research articles
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Showing results (21-30 of 82) with videos related to
Sort By:
Page
of 9
European Journal of Medical Genetics
|
June 30, 2023
A novel SLC5A6 homozygous variant in a family with multivitamin-dependent neurometabolic disorder: Phenotype expansion and long-term follow-up
Martino Montomoli, Annalisa Vetro, Flavia Tubili, et al.
JAMA Neurology
|
July 10, 2013
Mitochondrial encephalomyopathy due to a novel mutation in ACAD9
Caterina Garone, Maria Alice Donati, Michele Sacchini, et al.
Metabolic Brain Disease
|
June 4, 2017
The treatment of juvenile/adult GM1-gangliosidosis with Miglustat may reverse disease progression
Federica Deodato, Elena Procopio, Angelica Rampazzo, et al.
Molecular Genetics and Metabolism
|
July 5, 2017
A rare case of sterol-C4-methyl oxidase deficiency in a young Italian male: Biochemical and molecular characterization
Giulia Frisso, Monica Gelzo, Elena Procopio, et al.
International Journal of Cardiology
|
November 29, 2018
Clinical profile and outcome of cardiac involvement in MELAS syndrome
Alice Brambilla, Silvia Favilli, Iacopo Olivotto, et al.
BMC Medical Genomics
|
June 13, 2021
Bi-allelic variants in MTMR5/SBF1 cause Charcot-Marie-Tooth type 4B3 featuring mitochondrial dysfunction
Beatrice Berti, Giovanna Longo, Francesco Mari, et al.
Clinica Chimica Acta; International Journal of Clinical Chemistry
|
December 30, 2008
Rapid diagnostic testing procedures for lysosomal storage disorders: alpha-glucosidase and beta-galactosidase assays on dried blood spots
Nicoletta Gasparotto, Rosella Tomanin, Anna Chiara Frigo, et al.
Frontiers in Physiology
|
June 2, 2020
Advances in Stem Cell Modeling of Dystrophin-Associated Disease: Implications for the Wider World of Dilated Cardiomyopathy
Josè Manuel Pioner, Alessandra Fornaro, Raffaele Coppini, et al.
Metabolic Brain Disease
|
July 1, 2017
SSADH deficiency in an Italian family: a novel ALDH5A1 gene mutation affecting the succinic semialdehyde substrate binding site
Sara Leo, Concetta Capo, Bianca Maria Ciminelli, et al.
Molecular Genetics and Metabolism
|
December 3, 2019
Novel compound heterozygous pathogenic variants in nucleotide-binding protein like protein (NUBPL) cause leukoencephalopathy with multi-systemic involvement
Margherita Protasoni, Claudio Bruno, Maria Alice Donati, et al.
Page
of 9