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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 9, 2013
Autologous myoblast transplantation for oculopharyngeal muscular dystrophy: a phase I/IIa clinical study
Sophie Périé, Capucine Trollet, Vincent Mouly, et al.
Orphanet Journal of Rare Diseases
|
July 16, 2013
Partial lipodystrophy with severe insulin resistance and adult progeria Werner syndrome
Bruno Donadille, Pascal D'Anella, Martine Auclair, et al.
Archives of Neurology
|
August 19, 2007
Phenotypic study in 40 patients with dysferlin gene mutations: high frequency of atypical phenotypes
Karine Nguyen, Guillaume Bassez, Martin Krahn, et al.
Journal of Clinical Medicine
|
May 7, 2020
A Proteomics-Based Analysis Reveals Predictive Biological Patterns in Fabry Disease
Abdellah Tebani, Wladimir Mauhin, Lenaig Abily-Donval, et al.
Plos One
|
December 2, 2016
Correction: Cervical Spinal Cord Atrophy Profile in Adult SMN1-Linked SMA
Mohamed-Mounir El Mendili, Timothée Lenglet, Tanya Stojkovic, et al.
Neurology
|
April 12, 2015
Clinical and genetic spectrum in limb-girdle muscular dystrophy type 2E
Claudio Semplicini, John Vissing, Julia R Dahlqvist, et al.
Plos One
|
April 19, 2016
Cervical Spinal Cord Atrophy Profile in Adult SMN1-Linked SMA
Mohamed-Mounir El Mendili, Timothée Lenglet, Tanya Stojkovic, et al.
Neuromuscular Disorders : NMD
|
July 28, 2016
Cross-sectional retrospective study of muscle function in patients with glycogen storage disease type III
Valérie Decostre, Pascal Laforêt, Aleksandra Nadaj-Pakleza, et al.
Molecular Genetics and Metabolism
|
June 7, 2023
Hypersensitivity infusion-associated reactions induced by enzyme replacement therapy in a cohort of patients with late-onset Pompe disease: An experience from the French Pompe Registry
Lola E R Lessard, Céline Tard, Emmanuelle Salort-Campana, et al.
European Journal of Neurology
|
March 18, 2022
Macroglossia: A potentially severe complication of late-onset Pompe disease
Charlotte Dupé, Claire Lefeuvre, Guilhem Solé, et al.
Page
of 17
Search research articles
Search
Showing results (81-90 of 164) with videos related to
Sort By:
Page
of 17
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 9, 2013
Autologous myoblast transplantation for oculopharyngeal muscular dystrophy: a phase I/IIa clinical study
Sophie Périé, Capucine Trollet, Vincent Mouly, et al.
Orphanet Journal of Rare Diseases
|
July 16, 2013
Partial lipodystrophy with severe insulin resistance and adult progeria Werner syndrome
Bruno Donadille, Pascal D'Anella, Martine Auclair, et al.
Archives of Neurology
|
August 19, 2007
Phenotypic study in 40 patients with dysferlin gene mutations: high frequency of atypical phenotypes
Karine Nguyen, Guillaume Bassez, Martin Krahn, et al.
Journal of Clinical Medicine
|
May 7, 2020
A Proteomics-Based Analysis Reveals Predictive Biological Patterns in Fabry Disease
Abdellah Tebani, Wladimir Mauhin, Lenaig Abily-Donval, et al.
Plos One
|
December 2, 2016
Correction: Cervical Spinal Cord Atrophy Profile in Adult SMN1-Linked SMA
Mohamed-Mounir El Mendili, Timothée Lenglet, Tanya Stojkovic, et al.
Neurology
|
April 12, 2015
Clinical and genetic spectrum in limb-girdle muscular dystrophy type 2E
Claudio Semplicini, John Vissing, Julia R Dahlqvist, et al.
Plos One
|
April 19, 2016
Cervical Spinal Cord Atrophy Profile in Adult SMN1-Linked SMA
Mohamed-Mounir El Mendili, Timothée Lenglet, Tanya Stojkovic, et al.
Neuromuscular Disorders : NMD
|
July 28, 2016
Cross-sectional retrospective study of muscle function in patients with glycogen storage disease type III
Valérie Decostre, Pascal Laforêt, Aleksandra Nadaj-Pakleza, et al.
Molecular Genetics and Metabolism
|
June 7, 2023
Hypersensitivity infusion-associated reactions induced by enzyme replacement therapy in a cohort of patients with late-onset Pompe disease: An experience from the French Pompe Registry
Lola E R Lessard, Céline Tard, Emmanuelle Salort-Campana, et al.
European Journal of Neurology
|
March 18, 2022
Macroglossia: A potentially severe complication of late-onset Pompe disease
Charlotte Dupé, Claire Lefeuvre, Guilhem Solé, et al.
Page
of 17