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Plos Currents
|
January 19, 2013
Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational
Pauline McCormack, Simon Woods, Annemieke Aartsma-Rus, et al.
Acta Neuropathologica Communications
|
January 7, 2021
The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
Dominic Scaglioni, Francesco Catapano, Matthew Ellis, et al.
Neuromuscular Disorders : NMD
|
April 9, 2022
Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls
Francesco Muntoni, James Signorovitch, Gautam Sajeev, et al.
Neuromuscular Disorders : NMD
|
November 18, 2005
The differential gene expression profiles of proximal and distal muscle groups are altered in pre-pathological dysferlin-deficient mice
Maja von der Hagen, Steven H Laval, Lynsey M Cree, et al.
Neuromuscular Disorders : NMD
|
March 29, 2025
Late-onset multiple-acyl-CoA-dehydrogenase deficiency-like condition: a case series from the West of Scotland
Taylor Watson-Fargie, Autumn Coomber, Rachel Edwards, et al.
Journal of Neurology
|
January 19, 2013
A novel late-onset axial myopathy associated with mutations in the skeletal muscle ryanodine receptor (RYR1) gene
Sissel Løseth, Nicol C Voermans, Torberg Torbergsen, et al.
Plos One
|
June 27, 2025
Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy
Francesco Muntoni, James Signorovitch, Nathalie Goemans, et al.
Journal of Human Genetics
|
August 14, 2020
The clinical-phenotype continuum in DYNC1H1-related disorders-genomic profiling and proposal for a novel classification
Lena-Luise Becker, Hormos Salimi Dafsari, Jens Schallner, et al.
Epigenomics
|
November 20, 2020
Novel free-circulating and extracellular vesicle-derived miRNAs dysregulated in Duchenne muscular dystrophy
Francesco Catapano, Dominic Scaglioni, Kate Maresh, et al.
Plos One
|
July 10, 2024
Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study
Francesco Muntoni, James Signorovitch, Gautam Sajeev, et al.
Page
of 38
Search research articles
Search
Showing results (191-200 of 374) with videos related to
Sort By:
Page
of 38
Plos Currents
|
January 19, 2013
Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational
Pauline McCormack, Simon Woods, Annemieke Aartsma-Rus, et al.
Acta Neuropathologica Communications
|
January 7, 2021
The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
Dominic Scaglioni, Francesco Catapano, Matthew Ellis, et al.
Neuromuscular Disorders : NMD
|
April 9, 2022
Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls
Francesco Muntoni, James Signorovitch, Gautam Sajeev, et al.
Neuromuscular Disorders : NMD
|
November 18, 2005
The differential gene expression profiles of proximal and distal muscle groups are altered in pre-pathological dysferlin-deficient mice
Maja von der Hagen, Steven H Laval, Lynsey M Cree, et al.
Neuromuscular Disorders : NMD
|
March 29, 2025
Late-onset multiple-acyl-CoA-dehydrogenase deficiency-like condition: a case series from the West of Scotland
Taylor Watson-Fargie, Autumn Coomber, Rachel Edwards, et al.
Journal of Neurology
|
January 19, 2013
A novel late-onset axial myopathy associated with mutations in the skeletal muscle ryanodine receptor (RYR1) gene
Sissel Løseth, Nicol C Voermans, Torberg Torbergsen, et al.
Plos One
|
June 27, 2025
Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy
Francesco Muntoni, James Signorovitch, Nathalie Goemans, et al.
Journal of Human Genetics
|
August 14, 2020
The clinical-phenotype continuum in DYNC1H1-related disorders-genomic profiling and proposal for a novel classification
Lena-Luise Becker, Hormos Salimi Dafsari, Jens Schallner, et al.
Epigenomics
|
November 20, 2020
Novel free-circulating and extracellular vesicle-derived miRNAs dysregulated in Duchenne muscular dystrophy
Francesco Catapano, Dominic Scaglioni, Kate Maresh, et al.
Plos One
|
July 10, 2024
Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study
Francesco Muntoni, James Signorovitch, Gautam Sajeev, et al.
Page
of 38