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The Lancet Regional Health. Europe
|
January 3, 2024
Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom
Vasantha Gowda, Mark Atherton, Archana Murugan, et al.
Human Mutation
|
September 1, 2018
STAC3 variants cause a congenital myopathy with distinctive dysmorphic features and malignant hyperthermia susceptibility
Irina T Zaharieva, Anna Sarkozy, Pinki Munot, et al.
Brain : a Journal of Neurology
|
March 25, 2024
RTN2 deficiency results in an autosomal recessive distal motor neuropathy with lower limb spasticity
Reza Maroofian, Payam Sarraf, Thomas J O'Brien, et al.
Acta Neuropathologica
|
December 25, 2016
Dihydropyridine receptor (DHPR, CACNA1S) congenital myopathy
Vanessa Schartner, Norma B Romero, Sandra Donkervoort, et al.
Brain : a Journal of Neurology
|
June 11, 2025
Deciphering DST-associated disorders: biallelic variants affecting DST-b cause a congenital myopathy
Maureen Jacob, Heike Kölbel, Philip Harrer, et al.
Nature Communications
|
April 28, 2022
Heterozygous frameshift variants in HNRNPA2B1 cause early-onset oculopharyngeal muscular dystrophy
Hong Joo Kim, Payam Mohassel, Sandra Donkervoort, et al.
The Lancet. Neurology
|
July 19, 2025
Acute-onset axonal neuropathy following infection in children with biallelic RCC1 variants: a case series
J Robert Harkness, John H McDermott, Shea Marsden, et al.
Medrxiv : the Preprint Server for Health Sciences
|
April 8, 2024
The recurrent deep intronic pseudoexon-inducing variant <i>COL6A1</i> c.930+189C>T results in a consistently severe phenotype of COL6-related dystrophy: Towards clinical trial readiness for splice-modulating therapy
A Reghan Foley, Véronique Bolduc, Fady Guirguis, et al.
Brain : a Journal of Neurology
|
April 3, 2025
Characterization of severe COL6-related dystrophy due to the recurrent variant COL6A1 c.930+189C>T
A Reghan Foley, Véronique Bolduc, Fady Guirguis, et al.
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Showing results (51-60 of 59) with videos related to
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You have reached the last page of results.
This site can display upto 59 results.
The Lancet Regional Health. Europe
|
January 3, 2024
Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom
Vasantha Gowda, Mark Atherton, Archana Murugan, et al.
Human Mutation
|
September 1, 2018
STAC3 variants cause a congenital myopathy with distinctive dysmorphic features and malignant hyperthermia susceptibility
Irina T Zaharieva, Anna Sarkozy, Pinki Munot, et al.
Brain : a Journal of Neurology
|
March 25, 2024
RTN2 deficiency results in an autosomal recessive distal motor neuropathy with lower limb spasticity
Reza Maroofian, Payam Sarraf, Thomas J O'Brien, et al.
Acta Neuropathologica
|
December 25, 2016
Dihydropyridine receptor (DHPR, CACNA1S) congenital myopathy
Vanessa Schartner, Norma B Romero, Sandra Donkervoort, et al.
Brain : a Journal of Neurology
|
June 11, 2025
Deciphering DST-associated disorders: biallelic variants affecting DST-b cause a congenital myopathy
Maureen Jacob, Heike Kölbel, Philip Harrer, et al.
Nature Communications
|
April 28, 2022
Heterozygous frameshift variants in HNRNPA2B1 cause early-onset oculopharyngeal muscular dystrophy
Hong Joo Kim, Payam Mohassel, Sandra Donkervoort, et al.
The Lancet. Neurology
|
July 19, 2025
Acute-onset axonal neuropathy following infection in children with biallelic RCC1 variants: a case series
J Robert Harkness, John H McDermott, Shea Marsden, et al.
Medrxiv : the Preprint Server for Health Sciences
|
April 8, 2024
The recurrent deep intronic pseudoexon-inducing variant <i>COL6A1</i> c.930+189C>T results in a consistently severe phenotype of COL6-related dystrophy: Towards clinical trial readiness for splice-modulating therapy
A Reghan Foley, Véronique Bolduc, Fady Guirguis, et al.
Brain : a Journal of Neurology
|
April 3, 2025
Characterization of severe COL6-related dystrophy due to the recurrent variant COL6A1 c.930+189C>T
A Reghan Foley, Véronique Bolduc, Fady Guirguis, et al.
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of 6