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Medrxiv : the Preprint Server for Health Sciences
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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.
Nature Genetics
|
July 2, 2021
Pathogenic SPTBN1 variants cause an autosomal dominant neurodevelopmental syndrome
Margot A Cousin, Blake A Creighton, Keith A Breau, et al.
Brain : a Journal of Neurology
|
September 26, 2020
Defining the clinical, molecular and imaging spectrum of adaptor protein complex 4-associated hereditary spastic paraplegia
Darius Ebrahimi-Fakhari, Julian Teinert, Robert Behne, et al.
The Journal of Clinical Investigation
|
September 18, 2025
PTBP1 variants displaying altered nucleocytoplasmic distribution are responsible for a neurodevelopmental disorder with skeletal dysplasia
Aymeric Masson, Julien Paccaud, Martina Orefice, et al.
American Journal of Human Genetics
|
April 17, 2025
Bi-allelic pathogenic variants in TRMT1 disrupt tRNA modification and induce a neurodevelopmental disorder
Stephanie Efthymiou, Cailyn P Leo, Chenghong Deng, et al.
The Journal of Clinical Endocrinology and Metabolism
|
October 2, 2020
Endocrine and Growth Abnormalities in 4H Leukodystrophy Caused by Variants in POLR3A, POLR3B, and POLR1C
Félixe Pelletier, Stefanie Perrier, Ferdy K Cayami, et al.
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Search research articles
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Showing results (171-180 of 177) with videos related to
Sort By:
Page
of 18
You have reached the last page of results.
This site can display upto 177 results.
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.
Nature Genetics
|
July 2, 2021
Pathogenic SPTBN1 variants cause an autosomal dominant neurodevelopmental syndrome
Margot A Cousin, Blake A Creighton, Keith A Breau, et al.
Brain : a Journal of Neurology
|
September 26, 2020
Defining the clinical, molecular and imaging spectrum of adaptor protein complex 4-associated hereditary spastic paraplegia
Darius Ebrahimi-Fakhari, Julian Teinert, Robert Behne, et al.
The Journal of Clinical Investigation
|
September 18, 2025
PTBP1 variants displaying altered nucleocytoplasmic distribution are responsible for a neurodevelopmental disorder with skeletal dysplasia
Aymeric Masson, Julien Paccaud, Martina Orefice, et al.
American Journal of Human Genetics
|
April 17, 2025
Bi-allelic pathogenic variants in TRMT1 disrupt tRNA modification and induce a neurodevelopmental disorder
Stephanie Efthymiou, Cailyn P Leo, Chenghong Deng, et al.
The Journal of Clinical Endocrinology and Metabolism
|
October 2, 2020
Endocrine and Growth Abnormalities in 4H Leukodystrophy Caused by Variants in POLR3A, POLR3B, and POLR1C
Félixe Pelletier, Stefanie Perrier, Ferdy K Cayami, et al.
Page
of 18