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Human Molecular Genetics
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January 6, 2017
Structural analysis of pathogenic mutations in the DYRK1A gene in patients with developmental disorders
Jochem M G Evers, Roman A Laskowski, Marta Bertolli, et al.
Journal of Neurology, Neurosurgery, and Psychiatry
|
December 6, 2014
Validation of genetic modifiers for Duchenne muscular dystrophy: a multicentre study assessing SPP1 and LTBP4 variants
Janneke C van den Bergen, Monika Hiller, Stefan Böhringer, et al.
Neurology
|
May 26, 2022
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease
Mazen M Dimachkie, Richard J Barohn, Barry Byrne, et al.
Journal of Medical Genetics
|
June 28, 2019
SMCHD1 mutation spectrum for facioscapulohumeral muscular dystrophy type 2 (FSHD2) and Bosma arhinia microphthalmia syndrome (BAMS) reveals disease-specific localisation of variants in the ATPase domain
Richard J L F Lemmers, Nienke van der Stoep, Patrick J van der Vliet, et al.
The Lancet. Neurology
|
August 29, 2009
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
Maria Kinali, Virginia Arechavala-Gomeza, Lucy Feng, et al.
Journal of Neuromuscular Diseases
|
February 11, 2020
Improved Criteria for the Classification of Titin Variants in Inherited Skeletal Myopathies
Marco Savarese, Mridul Johari, Katherine Johnson, et al.
Muscle & Nerve
|
January 25, 2019
Revised upper limb module for spinal muscular atrophy: 12 month changes
Maria Carmela Pera, Giorgia Coratti, Elena S Mazzone, et al.
Neurology. Genetics
|
November 3, 2025
Motor Function in Limb-Girdle Muscular Dystrophy R1/2A: Validation of Clinical Outcome Assessments for Clinical Care and Trial Readiness
Meredith K James, Megan A Iammarino, Natalie F Reash, et al.
Scientific Reports
|
February 9, 2024
Imaging mass cytometry analysis of Becker muscular dystrophy muscle samples reveals different stages of muscle degeneration
Patricia Piñol-Jurado, José Verdú-Díaz, Esther Fernández-Simón, et al.
Neuromuscular Disorders : NMD
|
September 13, 2023
Muscle magnetic resonance imaging of a large cohort of distal hereditary motor neuropathies reveals characteristic features useful for diagnosis
Diana Esteller, Jasper Morrow, Jorge Alonso-Pérez, et al.
Page
of 38
Search research articles
Search
Showing results (271-280 of 374) with videos related to
Sort By:
Page
of 38
Human Molecular Genetics
|
January 6, 2017
Structural analysis of pathogenic mutations in the DYRK1A gene in patients with developmental disorders
Jochem M G Evers, Roman A Laskowski, Marta Bertolli, et al.
Journal of Neurology, Neurosurgery, and Psychiatry
|
December 6, 2014
Validation of genetic modifiers for Duchenne muscular dystrophy: a multicentre study assessing SPP1 and LTBP4 variants
Janneke C van den Bergen, Monika Hiller, Stefan Böhringer, et al.
Neurology
|
May 26, 2022
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease
Mazen M Dimachkie, Richard J Barohn, Barry Byrne, et al.
Journal of Medical Genetics
|
June 28, 2019
SMCHD1 mutation spectrum for facioscapulohumeral muscular dystrophy type 2 (FSHD2) and Bosma arhinia microphthalmia syndrome (BAMS) reveals disease-specific localisation of variants in the ATPase domain
Richard J L F Lemmers, Nienke van der Stoep, Patrick J van der Vliet, et al.
The Lancet. Neurology
|
August 29, 2009
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
Maria Kinali, Virginia Arechavala-Gomeza, Lucy Feng, et al.
Journal of Neuromuscular Diseases
|
February 11, 2020
Improved Criteria for the Classification of Titin Variants in Inherited Skeletal Myopathies
Marco Savarese, Mridul Johari, Katherine Johnson, et al.
Muscle & Nerve
|
January 25, 2019
Revised upper limb module for spinal muscular atrophy: 12 month changes
Maria Carmela Pera, Giorgia Coratti, Elena S Mazzone, et al.
Neurology. Genetics
|
November 3, 2025
Motor Function in Limb-Girdle Muscular Dystrophy R1/2A: Validation of Clinical Outcome Assessments for Clinical Care and Trial Readiness
Meredith K James, Megan A Iammarino, Natalie F Reash, et al.
Scientific Reports
|
February 9, 2024
Imaging mass cytometry analysis of Becker muscular dystrophy muscle samples reveals different stages of muscle degeneration
Patricia Piñol-Jurado, José Verdú-Díaz, Esther Fernández-Simón, et al.
Neuromuscular Disorders : NMD
|
September 13, 2023
Muscle magnetic resonance imaging of a large cohort of distal hereditary motor neuropathies reveals characteristic features useful for diagnosis
Diana Esteller, Jasper Morrow, Jorge Alonso-Pérez, et al.
Page
of 38