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Journal of Inherited Metabolic Disease
|
March 24, 2012
Long-term bone mineral density response to enzyme replacement therapy in a retrospective pediatric cohort of Gaucher patients
Giovanni Ciana, Laura Deroma, Anna Martina Franzil, et al.
Plos One
|
November 15, 2014
Characterization of a spontaneous novel mutation in the NPC2 gene in a cat affected by Niemann Pick type C disease
Stefania Zampieri, Ezio Bianchi, Carlo Cantile, et al.
Human Mutation
|
July 13, 2005
Functional in vitro characterization of 14 SMPD1 mutations identified in Italian patients affected by Niemann Pick Type B disease
Andrea Dardis, Stefania Zampieri, Mirella Filocamo, et al.
Molecular Genetics and Metabolism Reports
|
October 18, 2021
Focal hepatic lesions in acid sphingomyelinase deficiency: Differential diagnosis between foamy macrophages aggregates and malignancy
Annalisa Sechi, Alessandro Vit, Claudio Avellini, et al.
European Journal of Pediatrics
|
January 8, 2014
Enzyme replacement therapy in juvenile glycogenosis type II: a longitudinal study
Laura Deroma, Mattia Guerra, Annalisa Sechi, et al.
Pharmacoepidemiology and Drug Safety
|
February 7, 2015
Results from a 9-year Intensive Safety Surveillance Scheme (IS(3) ) in miglustat (Zavesca(®) )-treated patients
Monika Brand, Audrey Muller, Jonathan Alsop, et al.
Molecular Genetics and Metabolism
|
November 2, 2010
Molecular characterization of a new deletion of the GBA1 gene due to an inter Alu recombination event
Monica Cozar, Bruno Bembi, Silvia Dominissini, et al.
JIMD Reports
|
February 23, 2013
Did the temporary shortage in supply of imiglucerase have clinical consequences? Retrospective observational study on 34 italian Gaucher type I patients
Laura Deroma, Annalisa Sechi, Andrea Dardis, et al.
FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology
|
May 29, 2015
Role of LIMP-2 in the intracellular trafficking of β-glucosidase in different human cellular models
Erika Malini, Stefania Zampieri, Marta Deganuto, et al.
Pharmacoepidemiology and Drug Safety
|
June 10, 2009
Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme
Carla E M Hollak, Derralynn Hughes, Ivo N van Schaik, et al.
Page
of 11
Search research articles
Search
Showing results (11-20 of 101) with videos related to
Sort By:
Page
of 11
Journal of Inherited Metabolic Disease
|
March 24, 2012
Long-term bone mineral density response to enzyme replacement therapy in a retrospective pediatric cohort of Gaucher patients
Giovanni Ciana, Laura Deroma, Anna Martina Franzil, et al.
Plos One
|
November 15, 2014
Characterization of a spontaneous novel mutation in the NPC2 gene in a cat affected by Niemann Pick type C disease
Stefania Zampieri, Ezio Bianchi, Carlo Cantile, et al.
Human Mutation
|
July 13, 2005
Functional in vitro characterization of 14 SMPD1 mutations identified in Italian patients affected by Niemann Pick Type B disease
Andrea Dardis, Stefania Zampieri, Mirella Filocamo, et al.
Molecular Genetics and Metabolism Reports
|
October 18, 2021
Focal hepatic lesions in acid sphingomyelinase deficiency: Differential diagnosis between foamy macrophages aggregates and malignancy
Annalisa Sechi, Alessandro Vit, Claudio Avellini, et al.
European Journal of Pediatrics
|
January 8, 2014
Enzyme replacement therapy in juvenile glycogenosis type II: a longitudinal study
Laura Deroma, Mattia Guerra, Annalisa Sechi, et al.
Pharmacoepidemiology and Drug Safety
|
February 7, 2015
Results from a 9-year Intensive Safety Surveillance Scheme (IS(3) ) in miglustat (Zavesca(®) )-treated patients
Monika Brand, Audrey Muller, Jonathan Alsop, et al.
Molecular Genetics and Metabolism
|
November 2, 2010
Molecular characterization of a new deletion of the GBA1 gene due to an inter Alu recombination event
Monica Cozar, Bruno Bembi, Silvia Dominissini, et al.
JIMD Reports
|
February 23, 2013
Did the temporary shortage in supply of imiglucerase have clinical consequences? Retrospective observational study on 34 italian Gaucher type I patients
Laura Deroma, Annalisa Sechi, Andrea Dardis, et al.
FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology
|
May 29, 2015
Role of LIMP-2 in the intracellular trafficking of β-glucosidase in different human cellular models
Erika Malini, Stefania Zampieri, Marta Deganuto, et al.
Pharmacoepidemiology and Drug Safety
|
June 10, 2009
Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme
Carla E M Hollak, Derralynn Hughes, Ivo N van Schaik, et al.
Page
of 11