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Human Molecular Genetics
|
October 4, 2017
Defective mitochondrial rRNA methyltransferase MRM2 causes MELAS-like clinical syndrome
Caterina Garone, Aaron R D'Souza, Cristina Dallabona, et al.
Journal of Medical Genetics
|
June 17, 2021
Learning from massive testing of mitochondrial disorders: UPD explaining unorthodox transmission
Deborah Tolomeo, Anna Rubegni, Claudia Nesti, et al.
Frontiers in Neurology
|
October 22, 2021
The Role of Muscle Biopsy in Diagnostic Process of Infant Hypotonia: From Clinical Classification to the Genetic Outcome
Marco Veneruso, Chiara Fiorillo, Paolo Broda, et al.
Human Gene Therapy
|
February 6, 2015
Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial
Rita Ferla, Pamela Claudiani, Marco Savarese, et al.
Human Mutation
|
May 26, 2017
Recessive mutations in MSTO1 cause mitochondrial dynamics impairment, leading to myopathy and ataxia
Alessia Nasca, Chiara Scotton, Irina Zaharieva, et al.
Molecular Genetics and Metabolism
|
April 12, 2026
Over ten years of newborn screening for LSDs in Tuscany (Italy): Epidemiology, novel variants, and the pseudodeficiency burden
Sabrina Malvagia, Marta Daniotti, Rodolfo Tonin, et al.
Orphanet Journal of Rare Diseases
|
February 10, 2018
Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy
Rossella Parini, Paola De Lorenzo, Andrea Dardis, et al.
Brain : a Journal of Neurology
|
August 23, 2015
ALDH18A1 gene mutations cause dominant spastic paraplegia SPG9: loss of function effect and plausibility of a dominant negative mechanism
Emanuele Panza, Juan M Escamilla-Honrubia, Clara Marco-Marín, et al.
Human Mutation
|
May 18, 2004
Molecular and functional analysis of SUMF1 mutations in multiple sulfatase deficiency
Maria Pia Cosma, Stefano Pepe, Giancarlo Parenti, et al.
European Journal of Pediatrics
|
May 6, 2022
Neurological assessment of newborns with spinal muscular atrophy identified through neonatal screening
Marika Pane, Maria Alice Donati, Costanza Cutrona, et al.
Page
of 12
Search research articles
Search
Showing results (71-80 of 117) with videos related to
Sort By:
Page
of 12
Human Molecular Genetics
|
October 4, 2017
Defective mitochondrial rRNA methyltransferase MRM2 causes MELAS-like clinical syndrome
Caterina Garone, Aaron R D'Souza, Cristina Dallabona, et al.
Journal of Medical Genetics
|
June 17, 2021
Learning from massive testing of mitochondrial disorders: UPD explaining unorthodox transmission
Deborah Tolomeo, Anna Rubegni, Claudia Nesti, et al.
Frontiers in Neurology
|
October 22, 2021
The Role of Muscle Biopsy in Diagnostic Process of Infant Hypotonia: From Clinical Classification to the Genetic Outcome
Marco Veneruso, Chiara Fiorillo, Paolo Broda, et al.
Human Gene Therapy
|
February 6, 2015
Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial
Rita Ferla, Pamela Claudiani, Marco Savarese, et al.
Human Mutation
|
May 26, 2017
Recessive mutations in MSTO1 cause mitochondrial dynamics impairment, leading to myopathy and ataxia
Alessia Nasca, Chiara Scotton, Irina Zaharieva, et al.
Molecular Genetics and Metabolism
|
April 12, 2026
Over ten years of newborn screening for LSDs in Tuscany (Italy): Epidemiology, novel variants, and the pseudodeficiency burden
Sabrina Malvagia, Marta Daniotti, Rodolfo Tonin, et al.
Orphanet Journal of Rare Diseases
|
February 10, 2018
Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy
Rossella Parini, Paola De Lorenzo, Andrea Dardis, et al.
Brain : a Journal of Neurology
|
August 23, 2015
ALDH18A1 gene mutations cause dominant spastic paraplegia SPG9: loss of function effect and plausibility of a dominant negative mechanism
Emanuele Panza, Juan M Escamilla-Honrubia, Clara Marco-Marín, et al.
Human Mutation
|
May 18, 2004
Molecular and functional analysis of SUMF1 mutations in multiple sulfatase deficiency
Maria Pia Cosma, Stefano Pepe, Giancarlo Parenti, et al.
European Journal of Pediatrics
|
May 6, 2022
Neurological assessment of newborns with spinal muscular atrophy identified through neonatal screening
Marika Pane, Maria Alice Donati, Costanza Cutrona, et al.
Page
of 12