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Human Mutation
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October 2, 2010
Identification and characterization of 15 novel GALC gene mutations causing Krabbe disease
Barbara Tappino, Roberta Biancheri, Matthew Mort, et al.
Molecular Genetics and Metabolism
|
November 14, 2017
Corrigendum to "Cause of death in patients with chronic visceral and chronic neurovisceral acid sphingomyelinase deficiency (Niemann-Pick disease type B and B variant): Literature review and report of new cases" [Mol. Genet. Metab. 118 (2016) 206-213]
David Cassiman, Seymour Packman, Bruno Bembi, et al.
Molecular Genetics and Metabolism
|
May 21, 2016
Cause of death in patients with chronic visceral and chronic neurovisceral acid sphingomyelinase deficiency (Niemann-Pick disease type B and B variant): Literature review and report of new cases
David Cassiman, Seymour Packman, Bruno Bembi, et al.
American Journal of Hematology
|
November 2, 2017
Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS)
Ari Zimran, Nadia Belmatoug, Bruno Bembi, et al.
Minerva Anestesiologica
|
December 15, 2017
Facing up to limits: a lesson from the Charlie Gard case
Alberto Giannini, Paola Cogo, Bruno Bembi, et al.
Clinica Chimica Acta; International Journal of Clinical Chemistry
|
January 22, 2016
Comprehensive Evaluation of Plasma 7-Ketocholesterol and Cholestan-3β,5α,6β-Triol in an Italian Cohort of Patients Affected by Niemann-Pick Disease due to NPC1 and SMPD1 Mutations
Milena Romanello, Stefania Zampieri, Nadia Bortolotti, et al.
Radiology
|
November 24, 2005
Type B Niemann-Pick disease: findings at chest radiography, thin-section CT, and pulmonary function testing
David S Mendelson, Melissa P Wasserstein, Robert J Desnick, et al.
Clinica Chimica Acta; International Journal of Clinical Chemistry
|
July 24, 2012
First pilot newborn screening for four lysosomal storage diseases in an Italian region: identification and analysis of a putative causative mutation in the GBA gene
Silvia Paciotti, Emanuele Persichetti, Severo Pagliardini, et al.
Journal of Inherited Metabolic Disease
|
September 15, 2010
Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II
Bruno Bembi, Federica Edith Pisa, Marco Confalonieri, et al.
Pediatrics
|
July 16, 2008
A prospective, cross-sectional survey study of the natural history of Niemann-Pick disease type B
Margaret M McGovern, Melissa P Wasserstein, Roberto Giugliani, et al.
Page
of 11
Search research articles
Search
Showing results (61-70 of 101) with videos related to
Sort By:
Page
of 11
Human Mutation
|
October 2, 2010
Identification and characterization of 15 novel GALC gene mutations causing Krabbe disease
Barbara Tappino, Roberta Biancheri, Matthew Mort, et al.
Molecular Genetics and Metabolism
|
November 14, 2017
Corrigendum to "Cause of death in patients with chronic visceral and chronic neurovisceral acid sphingomyelinase deficiency (Niemann-Pick disease type B and B variant): Literature review and report of new cases" [Mol. Genet. Metab. 118 (2016) 206-213]
David Cassiman, Seymour Packman, Bruno Bembi, et al.
Molecular Genetics and Metabolism
|
May 21, 2016
Cause of death in patients with chronic visceral and chronic neurovisceral acid sphingomyelinase deficiency (Niemann-Pick disease type B and B variant): Literature review and report of new cases
David Cassiman, Seymour Packman, Bruno Bembi, et al.
American Journal of Hematology
|
November 2, 2017
Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS)
Ari Zimran, Nadia Belmatoug, Bruno Bembi, et al.
Minerva Anestesiologica
|
December 15, 2017
Facing up to limits: a lesson from the Charlie Gard case
Alberto Giannini, Paola Cogo, Bruno Bembi, et al.
Clinica Chimica Acta; International Journal of Clinical Chemistry
|
January 22, 2016
Comprehensive Evaluation of Plasma 7-Ketocholesterol and Cholestan-3β,5α,6β-Triol in an Italian Cohort of Patients Affected by Niemann-Pick Disease due to NPC1 and SMPD1 Mutations
Milena Romanello, Stefania Zampieri, Nadia Bortolotti, et al.
Radiology
|
November 24, 2005
Type B Niemann-Pick disease: findings at chest radiography, thin-section CT, and pulmonary function testing
David S Mendelson, Melissa P Wasserstein, Robert J Desnick, et al.
Clinica Chimica Acta; International Journal of Clinical Chemistry
|
July 24, 2012
First pilot newborn screening for four lysosomal storage diseases in an Italian region: identification and analysis of a putative causative mutation in the GBA gene
Silvia Paciotti, Emanuele Persichetti, Severo Pagliardini, et al.
Journal of Inherited Metabolic Disease
|
September 15, 2010
Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II
Bruno Bembi, Federica Edith Pisa, Marco Confalonieri, et al.
Pediatrics
|
July 16, 2008
A prospective, cross-sectional survey study of the natural history of Niemann-Pick disease type B
Margaret M McGovern, Melissa P Wasserstein, Roberto Giugliani, et al.
Page
of 11