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Orphanet Journal of Rare Diseases
|
September 30, 2020
Phenotypic diversity in an international Cure VCP Disease registry
Chiseko Ikenaga, Andrew R Findlay, Michelle Seiffert, et al.
Neuromuscular Disorders : NMD
|
November 25, 2025
287th ENMC international workshop: Harmonization and federated analysis of myotonic dystrophy registries to model heterogeneous disease trajectories. Hoofddorp, the Netherlands, 28-30 March 2025
Leandre A la Fontaine, Daniël van As, Guillaume Bassez, et al.
Muscle & Nerve
|
July 22, 2014
Prospective study of muscle cramps in Charcot-Marie-tooth disease
Nicholas E Johnson, Janet Sowden, Nuran Dilek, et al.
Bioanalysis
|
July 3, 2026
A simple, sensitive microsample LC-MS assay for quercetin and isorhamnetin in mouse and human plasma: application to EMIQ treatment in myotonic dystrophy type 1
Purnajai Srivijay Saravanan Vanaja, Subodh Mishra, John Douglas Cleary, et al.
Neurology. Genetics
|
July 15, 2026
Defining Haplosufficiency in Autosomal Recessive Limb-Girdle Muscular Dystrophy Using Molecular Markers in Disease Carriers
Alison Gaynor, Dove Enicks, Katherine Karam, et al.
Neurology
|
February 27, 2019
Effects of weakness of orofacial muscles on swallowing and communication in FSHD
Karlien Mul, Kiera N Berggren, Mattie Y Sills, et al.
Neuromuscular Disorders : NMD
|
August 6, 2014
Quality-of-life in Charcot-Marie-Tooth disease: the patient's perspective
Nicholas E Johnson, Chad R Heatwole, Nuran Dilek, et al.
Nature Reviews. Neurology
|
September 22, 2025
Myotonic dystrophy type 1: clinical diversity, molecular insights and therapeutic perspectives
Lisa Rahm, Melissa A Hale, Renée H L Raaijmakers, et al.
Muscle & Nerve
|
November 19, 2016
Physical function and mobility in children with congenital myotonic dystrophy
Evan M Pucillo, Deanna L Dibella, Man Hung, et al.
Muscle & Nerve
|
June 15, 2021
Long-term efficacy and safety of dichlorphenamide for treatment of primary periodic paralysis
Valeria A Sansone, Nicholas E Johnson, Michael G Hanna, et al.
Page
of 11
Search research articles
Search
Showing results (41-50 of 110) with videos related to
Sort By:
Page
of 11
Orphanet Journal of Rare Diseases
|
September 30, 2020
Phenotypic diversity in an international Cure VCP Disease registry
Chiseko Ikenaga, Andrew R Findlay, Michelle Seiffert, et al.
Neuromuscular Disorders : NMD
|
November 25, 2025
287th ENMC international workshop: Harmonization and federated analysis of myotonic dystrophy registries to model heterogeneous disease trajectories. Hoofddorp, the Netherlands, 28-30 March 2025
Leandre A la Fontaine, Daniël van As, Guillaume Bassez, et al.
Muscle & Nerve
|
July 22, 2014
Prospective study of muscle cramps in Charcot-Marie-tooth disease
Nicholas E Johnson, Janet Sowden, Nuran Dilek, et al.
Bioanalysis
|
July 3, 2026
A simple, sensitive microsample LC-MS assay for quercetin and isorhamnetin in mouse and human plasma: application to EMIQ treatment in myotonic dystrophy type 1
Purnajai Srivijay Saravanan Vanaja, Subodh Mishra, John Douglas Cleary, et al.
Neurology. Genetics
|
July 15, 2026
Defining Haplosufficiency in Autosomal Recessive Limb-Girdle Muscular Dystrophy Using Molecular Markers in Disease Carriers
Alison Gaynor, Dove Enicks, Katherine Karam, et al.
Neurology
|
February 27, 2019
Effects of weakness of orofacial muscles on swallowing and communication in FSHD
Karlien Mul, Kiera N Berggren, Mattie Y Sills, et al.
Neuromuscular Disorders : NMD
|
August 6, 2014
Quality-of-life in Charcot-Marie-Tooth disease: the patient's perspective
Nicholas E Johnson, Chad R Heatwole, Nuran Dilek, et al.
Nature Reviews. Neurology
|
September 22, 2025
Myotonic dystrophy type 1: clinical diversity, molecular insights and therapeutic perspectives
Lisa Rahm, Melissa A Hale, Renée H L Raaijmakers, et al.
Muscle & Nerve
|
November 19, 2016
Physical function and mobility in children with congenital myotonic dystrophy
Evan M Pucillo, Deanna L Dibella, Man Hung, et al.
Muscle & Nerve
|
June 15, 2021
Long-term efficacy and safety of dichlorphenamide for treatment of primary periodic paralysis
Valeria A Sansone, Nicholas E Johnson, Michael G Hanna, et al.
Page
of 11