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European Journal of Medical Genetics
|
February 15, 2012
A new lysosomal storage disorder resembling Morquio syndrome in sibs
Laurence Perrin, Odile Fenneteau, Brice Ilharreborde, et al.
Journal of Inherited Metabolic Disease
|
August 30, 2018
Late-onset Pompe disease in France: molecular features and epidemiology from a nationwide study
Claudio Semplicini, Pascaline Letard, Marie De Antonio, et al.
Molecular Genetics and Metabolism
|
July 16, 2023
Motor outcomes in patients with infantile and juvenile Pompe disease: Lessons from neurophysiological findings
Anaïs Brassier, Samia Pichard, Manuel Schiff, et al.
American Journal of Medical Genetics. Part A
|
January 5, 2011
Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece
Bénédicte Héron, Yann Mikaeloff, Roseline Froissart, et al.
Scientific Reports
|
November 5, 2016
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients
Elisa Masat, Pascal Laforêt, Marie De Antonio, et al.
Orphanet Journal of Rare Diseases
|
April 18, 2015
Natural disease history and characterisation of SUMF1 molecular defects in ten unrelated patients with multiple sulfatase deficiency
Frédérique Sabourdy, Lionel Mourey, Emmanuelle Le Trionnaire, et al.
Clinical Pharmacokinetics
|
August 22, 2018
Intra-monocyte Pharmacokinetics of Imiglucerase Supports a Possible Personalized Management of Gaucher Disease Type 1
Juliette Berger, Marie Vigan, Bruno Pereira, et al.
Brain : a Journal of Neurology
|
December 20, 2019
Homozygous GRN mutations: new phenotypes and new insights into pathological and molecular mechanisms
Vincent Huin, Mathieu Barbier, Armand Bottani, et al.
Cell Reports
|
June 28, 2018
Inhibition of Lysosome Membrane Recycling Causes Accumulation of Gangliosides that Contribute to Neurodegeneration
Maxime Boutry, Julien Branchu, Céline Lustremant, et al.
Birth Defects Research
|
September 7, 2021
Prenatal-onset of congenital neuronal ceroid lipofuscinosis with a novel CTSD mutation
Suzanne Chartier, Lucile Boutaud, Edouard Le Guillou, et al.
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of 9
Search research articles
Search
Showing results (61-70 of 86) with videos related to
Sort By:
Page
of 9
European Journal of Medical Genetics
|
February 15, 2012
A new lysosomal storage disorder resembling Morquio syndrome in sibs
Laurence Perrin, Odile Fenneteau, Brice Ilharreborde, et al.
Journal of Inherited Metabolic Disease
|
August 30, 2018
Late-onset Pompe disease in France: molecular features and epidemiology from a nationwide study
Claudio Semplicini, Pascaline Letard, Marie De Antonio, et al.
Molecular Genetics and Metabolism
|
July 16, 2023
Motor outcomes in patients with infantile and juvenile Pompe disease: Lessons from neurophysiological findings
Anaïs Brassier, Samia Pichard, Manuel Schiff, et al.
American Journal of Medical Genetics. Part A
|
January 5, 2011
Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece
Bénédicte Héron, Yann Mikaeloff, Roseline Froissart, et al.
Scientific Reports
|
November 5, 2016
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients
Elisa Masat, Pascal Laforêt, Marie De Antonio, et al.
Orphanet Journal of Rare Diseases
|
April 18, 2015
Natural disease history and characterisation of SUMF1 molecular defects in ten unrelated patients with multiple sulfatase deficiency
Frédérique Sabourdy, Lionel Mourey, Emmanuelle Le Trionnaire, et al.
Clinical Pharmacokinetics
|
August 22, 2018
Intra-monocyte Pharmacokinetics of Imiglucerase Supports a Possible Personalized Management of Gaucher Disease Type 1
Juliette Berger, Marie Vigan, Bruno Pereira, et al.
Brain : a Journal of Neurology
|
December 20, 2019
Homozygous GRN mutations: new phenotypes and new insights into pathological and molecular mechanisms
Vincent Huin, Mathieu Barbier, Armand Bottani, et al.
Cell Reports
|
June 28, 2018
Inhibition of Lysosome Membrane Recycling Causes Accumulation of Gangliosides that Contribute to Neurodegeneration
Maxime Boutry, Julien Branchu, Céline Lustremant, et al.
Birth Defects Research
|
September 7, 2021
Prenatal-onset of congenital neuronal ceroid lipofuscinosis with a novel CTSD mutation
Suzanne Chartier, Lucile Boutaud, Edouard Le Guillou, et al.
Page
of 9