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Genome Research
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March 26, 2025
Unraveling undiagnosed rare disease cases by HiFi long-read genome sequencing
Wouter Steyaert, Lydia Sagath, German Demidov, et al.
Medrxiv : the Preprint Server for Health Sciences
|
May 15, 2024
Unravelling undiagnosed rare disease cases by HiFi long-read genome sequencing
Wouter Steyaert, Lydia Sagath, German Demidov, et al.
Neurology
|
February 9, 2024
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial
Utkarsh J Dang, Jesse M Damsker, Michela Guglieri, et al.
JAMA Neurology
|
August 29, 2022
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial
Michela Guglieri, Paula R Clemens, Seth J Perlman, et al.
Brain : a Journal of Neurology
|
November 21, 2013
Treatable childhood neuronopathy caused by mutations in riboflavin transporter RFVT2
A Reghan Foley, Manoj P Menezes, Amelie Pandraud, 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.
Human Mutation
|
January 22, 2015
The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations
Catherine L Bladen, David Salgado, Soledad Monges, et al.
American Journal of Human Genetics
|
April 16, 2021
A form of muscular dystrophy associated with pathogenic variants in JAG2
Sandra Coppens, Alison M Barnard, Sanna Puusepp, et al.
Medrxiv : the Preprint Server for Health Sciences
|
July 16, 2025
Missense variants in <i>TUBA4A</i> cause myo-tubulinopathies
Mridul Johari, Chiara Folland, Yoshihiko Saito, et al.
Journal of Neurology, Neurosurgery, and Psychiatry
|
December 12, 2018
Muscle MRI in a large cohort of patients with oculopharyngeal muscular dystrophy
Alicia Alonso-Jimenez, Rosemarie H M J M Kroon, Aida Alejaldre-Monforte, et al.
Page
of 38
Search research articles
Search
Showing results (341-350 of 374) with videos related to
Sort By:
Page
of 38
Genome Research
|
March 26, 2025
Unraveling undiagnosed rare disease cases by HiFi long-read genome sequencing
Wouter Steyaert, Lydia Sagath, German Demidov, et al.
Medrxiv : the Preprint Server for Health Sciences
|
May 15, 2024
Unravelling undiagnosed rare disease cases by HiFi long-read genome sequencing
Wouter Steyaert, Lydia Sagath, German Demidov, et al.
Neurology
|
February 9, 2024
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial
Utkarsh J Dang, Jesse M Damsker, Michela Guglieri, et al.
JAMA Neurology
|
August 29, 2022
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial
Michela Guglieri, Paula R Clemens, Seth J Perlman, et al.
Brain : a Journal of Neurology
|
November 21, 2013
Treatable childhood neuronopathy caused by mutations in riboflavin transporter RFVT2
A Reghan Foley, Manoj P Menezes, Amelie Pandraud, 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.
Human Mutation
|
January 22, 2015
The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations
Catherine L Bladen, David Salgado, Soledad Monges, et al.
American Journal of Human Genetics
|
April 16, 2021
A form of muscular dystrophy associated with pathogenic variants in JAG2
Sandra Coppens, Alison M Barnard, Sanna Puusepp, et al.
Medrxiv : the Preprint Server for Health Sciences
|
July 16, 2025
Missense variants in <i>TUBA4A</i> cause myo-tubulinopathies
Mridul Johari, Chiara Folland, Yoshihiko Saito, et al.
Journal of Neurology, Neurosurgery, and Psychiatry
|
December 12, 2018
Muscle MRI in a large cohort of patients with oculopharyngeal muscular dystrophy
Alicia Alonso-Jimenez, Rosemarie H M J M Kroon, Aida Alejaldre-Monforte, et al.
Page
of 38