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HGG Advances
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July 18, 2022
Prevalence, parameters, and pathogenic mechanisms for splice-altering acceptor variants that disrupt the AG exclusion zone
Samantha J Bryen, Michaela Yuen, Himanshu Joshi, et al.
American Journal of Medical Genetics. Part A
|
June 22, 2021
L-carnitine supplementation for muscle weakness and fatigue in children with neurofibromatosis type 1: A Phase 2a clinical trial
Emily R Vasiljevski, Joshua Burns, Paula Bray, et al.
Molecular Genetics and Metabolism
|
August 4, 2022
The relationship between beta-ureidopropionase deficiency due to UPB1 variants and human phenotypes is uncertain
Sarah Righetti, Richard J N Allcock, Joy Yaplito-Lee, et al.
The Lancet Regional Health. Western Pacific
|
November 19, 2024
A contemporary analysis of the Australian clinical and genetic landscape of spinal muscular atrophy: a registry based study
Lakshmi Balaji, Robin Forbes, Anita Cairns, et al.
Neuromuscular Disorders : NMD
|
November 11, 2019
Importance of muscle biopsy to establish pathogenicity of DMD missense and splice variants
Hannah F Jones, Samantha J Bryen, Leigh B Waddell, et al.
Journal of Paediatrics and Child Health
|
April 12, 2024
Genomic multidisciplinary teams: A model for navigating genetic mainstreaming and precision medicine
Alan Ma, Timothy P Newing, Rosie O'Shea, et al.
Journal of Neurology, Neurosurgery, and Psychiatry
|
March 18, 2018
Nusinersen for SMA: expanded access programme
Michelle A Farrar, Hooi Ling Teoh, Kate A Carey, et al.
Human Mutation
|
December 22, 2017
Nemaline myopathy and distal arthrogryposis associated with an autosomal recessive TNNT3 splice variant
Sarah A Sandaradura, Adam Bournazos, Amali Mallawaarachchi, et al.
Journal of Cachexia, Sarcopenia and Muscle
|
August 15, 2025
High-Density Lipoprotein-Associated Cholesterol Abnormalities in a Clinical Outcomes Study of Dysferlin-Deficient Limb-Girdle Muscular Dystrophy Type R2
Zoe White, Laura Rufibach, Heather Gordish Dressman, et al.
Journal of Neurology
|
May 26, 2026
Identification of prognostic biomarkers in a large cohort of patients with LGMD R2
Carla F Bolano-Diaz, Jose Verdu-Diaz, Dan Hao, et al.
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of 5
Search research articles
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Showing results (11-20 of 47) with videos related to
Sort By:
Page
of 5
HGG Advances
|
July 18, 2022
Prevalence, parameters, and pathogenic mechanisms for splice-altering acceptor variants that disrupt the AG exclusion zone
Samantha J Bryen, Michaela Yuen, Himanshu Joshi, et al.
American Journal of Medical Genetics. Part A
|
June 22, 2021
L-carnitine supplementation for muscle weakness and fatigue in children with neurofibromatosis type 1: A Phase 2a clinical trial
Emily R Vasiljevski, Joshua Burns, Paula Bray, et al.
Molecular Genetics and Metabolism
|
August 4, 2022
The relationship between beta-ureidopropionase deficiency due to UPB1 variants and human phenotypes is uncertain
Sarah Righetti, Richard J N Allcock, Joy Yaplito-Lee, et al.
The Lancet Regional Health. Western Pacific
|
November 19, 2024
A contemporary analysis of the Australian clinical and genetic landscape of spinal muscular atrophy: a registry based study
Lakshmi Balaji, Robin Forbes, Anita Cairns, et al.
Neuromuscular Disorders : NMD
|
November 11, 2019
Importance of muscle biopsy to establish pathogenicity of DMD missense and splice variants
Hannah F Jones, Samantha J Bryen, Leigh B Waddell, et al.
Journal of Paediatrics and Child Health
|
April 12, 2024
Genomic multidisciplinary teams: A model for navigating genetic mainstreaming and precision medicine
Alan Ma, Timothy P Newing, Rosie O'Shea, et al.
Journal of Neurology, Neurosurgery, and Psychiatry
|
March 18, 2018
Nusinersen for SMA: expanded access programme
Michelle A Farrar, Hooi Ling Teoh, Kate A Carey, et al.
Human Mutation
|
December 22, 2017
Nemaline myopathy and distal arthrogryposis associated with an autosomal recessive TNNT3 splice variant
Sarah A Sandaradura, Adam Bournazos, Amali Mallawaarachchi, et al.
Journal of Cachexia, Sarcopenia and Muscle
|
August 15, 2025
High-Density Lipoprotein-Associated Cholesterol Abnormalities in a Clinical Outcomes Study of Dysferlin-Deficient Limb-Girdle Muscular Dystrophy Type R2
Zoe White, Laura Rufibach, Heather Gordish Dressman, et al.
Journal of Neurology
|
May 26, 2026
Identification of prognostic biomarkers in a large cohort of patients with LGMD R2
Carla F Bolano-Diaz, Jose Verdu-Diaz, Dan Hao, et al.
Page
of 5