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Acta Neuropathologica
|
December 17, 2008
"Necklace" fibers, a new histological marker of late-onset MTM1-related centronuclear myopathy
Jorge A Bevilacqua, Marc Bitoun, Valérie Biancalana, et al.
Orphanet Journal of Rare Diseases
|
February 14, 2025
Real-world evidence for Pompe disease remains fragmented. Comment on "A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem" by Klein et al
Michelle E Kruijshaar, Tiffany House, Benedikt Schoser, et al.
Archives of Cardiovascular Diseases
|
October 22, 2013
Brugada syndrome and abnormal splicing of SCN5A in myotonic dystrophy type 1
Karim Wahbi, Vincent Algalarrondo, Henri Marc Bécane, et al.
Nature Genetics
|
October 18, 2005
Mutations in dynamin 2 cause dominant centronuclear myopathy
Marc Bitoun, Svetlana Maugenre, Pierre-Yves Jeannet, et al.
Molecular Genetics and Metabolism
|
September 11, 2017
Long term longitudinal study of muscle function in patients with glycogen storage disease type IIIa
Valérie Decostre, Pascal Laforêt, Marie De Antonio, et al.
Circulation. Cardiovascular Genetics
|
June 15, 2017
Association Between Mutation Size and Cardiac Involvement in Myotonic Dystrophy Type 1: An Analysis of the DM1-Heart Registry
Caroline Chong-Nguyen, Karim Wahbi, Vincent Algalarrondo, et al.
Annals of Neurology
|
May 10, 2020
A New Glycogen Storage Disease Caused by a Dominant PYGM Mutation
Andoni Echaniz-Laguna, Xavière Lornage, Pascal Laforêt, et al.
Genetic Testing and Molecular Biomarkers
|
June 21, 2018
Genetic Mutations and Demographic, Clinical, and Morphological Aspects of Myofibrillar Myopathy in a French Cohort
Alzira Alves de Siqueira Carvalho, Emmanuele Lacene, Guy Brochier, et al.
Journal of Neuromuscular Diseases
|
April 5, 2021
Improved Cardiac Outcomes by Early Treatment with Angiotensin-Converting Enzyme Inhibitors in Becker Muscular Dystrophy
Caroline Stalens, Leslie Motté, Anthony Béhin, 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.
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of 17
Search research articles
Search
Showing results (91-100 of 164) with videos related to
Sort By:
Page
of 17
Acta Neuropathologica
|
December 17, 2008
"Necklace" fibers, a new histological marker of late-onset MTM1-related centronuclear myopathy
Jorge A Bevilacqua, Marc Bitoun, Valérie Biancalana, et al.
Orphanet Journal of Rare Diseases
|
February 14, 2025
Real-world evidence for Pompe disease remains fragmented. Comment on "A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem" by Klein et al
Michelle E Kruijshaar, Tiffany House, Benedikt Schoser, et al.
Archives of Cardiovascular Diseases
|
October 22, 2013
Brugada syndrome and abnormal splicing of SCN5A in myotonic dystrophy type 1
Karim Wahbi, Vincent Algalarrondo, Henri Marc Bécane, et al.
Nature Genetics
|
October 18, 2005
Mutations in dynamin 2 cause dominant centronuclear myopathy
Marc Bitoun, Svetlana Maugenre, Pierre-Yves Jeannet, et al.
Molecular Genetics and Metabolism
|
September 11, 2017
Long term longitudinal study of muscle function in patients with glycogen storage disease type IIIa
Valérie Decostre, Pascal Laforêt, Marie De Antonio, et al.
Circulation. Cardiovascular Genetics
|
June 15, 2017
Association Between Mutation Size and Cardiac Involvement in Myotonic Dystrophy Type 1: An Analysis of the DM1-Heart Registry
Caroline Chong-Nguyen, Karim Wahbi, Vincent Algalarrondo, et al.
Annals of Neurology
|
May 10, 2020
A New Glycogen Storage Disease Caused by a Dominant PYGM Mutation
Andoni Echaniz-Laguna, Xavière Lornage, Pascal Laforêt, et al.
Genetic Testing and Molecular Biomarkers
|
June 21, 2018
Genetic Mutations and Demographic, Clinical, and Morphological Aspects of Myofibrillar Myopathy in a French Cohort
Alzira Alves de Siqueira Carvalho, Emmanuele Lacene, Guy Brochier, et al.
Journal of Neuromuscular Diseases
|
April 5, 2021
Improved Cardiac Outcomes by Early Treatment with Angiotensin-Converting Enzyme Inhibitors in Becker Muscular Dystrophy
Caroline Stalens, Leslie Motté, Anthony Béhin, 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.
Page
of 17