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Italian Journal of Pediatrics
|
November 17, 2018
A new case report of severe mucopolysaccharidosis type VII: diagnosis, treatment with haematopoietic cell transplantation and prenatal diagnosis in a second pregnancy
Francesca Furlan, Attilio Rovelli, Miriam Rigoldi, et al.
Iscience
|
September 21, 2023
A GWAS in the pandemic epicenter highlights the severe COVID-19 risk locus introgressed by Neanderthals
Matteo Breno, Marina Noris, Nadia Rubis, et al.
Journal of Inherited Metabolic Disease
|
February 10, 2018
Hepatocellular carcinoma in Gaucher disease: an international case series
Martine Regenboog, Laura van Dussen, Joanne Verheij, et al.
Orphanet Journal of Rare Diseases
|
August 9, 2014
Genotype-phenotype correlation in Pompe disease, a step forward
Paola De Filippi, Kolsoum Saeidi, Sabrina Ravaglia, et al.
Journal of Inherited Metabolic Disease
|
February 18, 2020
Dietary lipids in glycogen storage disease type III: A systematic literature study, case studies, and future recommendations
Alessandro Rossi, Irene J Hoogeveen, Vanessa B Bastek, et al.
Human Mutation
|
April 17, 2009
Identification and molecular characterization of six novel mutations in the UDP-N-acetylglucosamine-1-phosphotransferase gamma subunit (GNPTG) gene in patients with mucolipidosis III gamma
Emanuele Persichetti, Nadia A Chuzhanova, Andrea Dardis, et al.
Molecular Genetics and Metabolism Reports
|
March 4, 2016
Enzymatic replacement therapy for Hunter disease: Up to 9 years experience with 17 patients
Rossella Parini, Miriam Rigoldi, Lucia Tedesco, et al.
Clinical Epigenetics
|
July 3, 2021
PRDX1 gene-related epi-cblC disease is a common type of inborn error of cobalamin metabolism with mono- or bi-allelic MMACHC epimutations
Catia Cavicchi, Abderrahim Oussalah, Silvia Falliano, et al.
Human Mutation
|
March 12, 2011
IDUA mutational profiling of a cohort of 102 European patients with mucopolysaccharidosis type I: identification and characterization of 35 novel α-L-iduronidase (IDUA) alleles
Francesca Bertola, Mirella Filocamo, Giorgio Casati, et al.
Orphanet Journal of Rare Diseases
|
July 26, 2018
Improving the diagnosis of cobalamin and related defects by genomic analysis, plus functional and structural assessment of novel variants
Sandra Brasil, Fátima Leal, Ana Vega, et al.
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of 4
Search research articles
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Showing results (21-30 of 35) with videos related to
Sort By:
Page
of 4
Italian Journal of Pediatrics
|
November 17, 2018
A new case report of severe mucopolysaccharidosis type VII: diagnosis, treatment with haematopoietic cell transplantation and prenatal diagnosis in a second pregnancy
Francesca Furlan, Attilio Rovelli, Miriam Rigoldi, et al.
Iscience
|
September 21, 2023
A GWAS in the pandemic epicenter highlights the severe COVID-19 risk locus introgressed by Neanderthals
Matteo Breno, Marina Noris, Nadia Rubis, et al.
Journal of Inherited Metabolic Disease
|
February 10, 2018
Hepatocellular carcinoma in Gaucher disease: an international case series
Martine Regenboog, Laura van Dussen, Joanne Verheij, et al.
Orphanet Journal of Rare Diseases
|
August 9, 2014
Genotype-phenotype correlation in Pompe disease, a step forward
Paola De Filippi, Kolsoum Saeidi, Sabrina Ravaglia, et al.
Journal of Inherited Metabolic Disease
|
February 18, 2020
Dietary lipids in glycogen storage disease type III: A systematic literature study, case studies, and future recommendations
Alessandro Rossi, Irene J Hoogeveen, Vanessa B Bastek, et al.
Human Mutation
|
April 17, 2009
Identification and molecular characterization of six novel mutations in the UDP-N-acetylglucosamine-1-phosphotransferase gamma subunit (GNPTG) gene in patients with mucolipidosis III gamma
Emanuele Persichetti, Nadia A Chuzhanova, Andrea Dardis, et al.
Molecular Genetics and Metabolism Reports
|
March 4, 2016
Enzymatic replacement therapy for Hunter disease: Up to 9 years experience with 17 patients
Rossella Parini, Miriam Rigoldi, Lucia Tedesco, et al.
Clinical Epigenetics
|
July 3, 2021
PRDX1 gene-related epi-cblC disease is a common type of inborn error of cobalamin metabolism with mono- or bi-allelic MMACHC epimutations
Catia Cavicchi, Abderrahim Oussalah, Silvia Falliano, et al.
Human Mutation
|
March 12, 2011
IDUA mutational profiling of a cohort of 102 European patients with mucopolysaccharidosis type I: identification and characterization of 35 novel α-L-iduronidase (IDUA) alleles
Francesca Bertola, Mirella Filocamo, Giorgio Casati, et al.
Orphanet Journal of Rare Diseases
|
July 26, 2018
Improving the diagnosis of cobalamin and related defects by genomic analysis, plus functional and structural assessment of novel variants
Sandra Brasil, Fátima Leal, Ana Vega, et al.
Page
of 4