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Science Translational Medicine
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April 21, 2017
Improving genetic diagnosis in Mendelian disease with transcriptome sequencing
Beryl B Cummings, Jamie L Marshall, Taru Tukiainen, et al.
Annals of Clinical and Translational Neurology
|
December 31, 2025
Whole-Body Pattern of Muscle Degeneration and Progression in Sarcoglycanopathies
Laura Costa-Comellas, Mauro Monforte, Angel Sanchez-Montañez, et al.
American Journal of Human Genetics
|
November 20, 2020
Pathogenic Variants in the Myosin Chaperone UNC-45B Cause Progressive Myopathy with Eccentric Cores
Sandra Donkervoort, Carl E Kutzner, Ying Hu, et al.
Annals of Neurology
|
February 12, 2021
Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale
Marni B Jacobs, Meredoith K James, Linda P Lowes, et al.
Frontiers in Neurology
|
April 1, 2022
Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach
Anna G Mayhew, Meredith K James, Ursula Moore, et al.
Contemporary Clinical Trials
|
April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
Michela Guglieri, Kate Bushby, Michael P McDermott, et al.
Neurology. Genetics
|
August 17, 2023
Clinical Classification of Variants in the Valosin-Containing Protein Gene Associated With Multisystem Proteinopathy
Marianela Schiava, Chiseko Ikenaga, Ana Topf, et al.
Nature Medicine
|
June 1, 2021
Childhood amyotrophic lateral sclerosis caused by excess sphingolipid synthesis
Payam Mohassel, Sandra Donkervoort, Museer A Lone, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
July 1, 2025
Bi-allelic loss-of-function variants in POC5 cause a syndromic retinal, endocrine, and neuromuscular ciliopathy
Anneke T Vulto-van Silfhout, Ingrid M Jazet, Suzanne Yzer, et al.
Trials
|
May 26, 2018
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial
Rebecca A Crow, Kimberly A Hart, Michael P McDermott, et al.
Page
of 38
Search research articles
Search
Showing results (331-340 of 374) with videos related to
Sort By:
Page
of 38
Science Translational Medicine
|
April 21, 2017
Improving genetic diagnosis in Mendelian disease with transcriptome sequencing
Beryl B Cummings, Jamie L Marshall, Taru Tukiainen, et al.
Annals of Clinical and Translational Neurology
|
December 31, 2025
Whole-Body Pattern of Muscle Degeneration and Progression in Sarcoglycanopathies
Laura Costa-Comellas, Mauro Monforte, Angel Sanchez-Montañez, et al.
American Journal of Human Genetics
|
November 20, 2020
Pathogenic Variants in the Myosin Chaperone UNC-45B Cause Progressive Myopathy with Eccentric Cores
Sandra Donkervoort, Carl E Kutzner, Ying Hu, et al.
Annals of Neurology
|
February 12, 2021
Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale
Marni B Jacobs, Meredoith K James, Linda P Lowes, et al.
Frontiers in Neurology
|
April 1, 2022
Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach
Anna G Mayhew, Meredith K James, Ursula Moore, et al.
Contemporary Clinical Trials
|
April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
Michela Guglieri, Kate Bushby, Michael P McDermott, et al.
Neurology. Genetics
|
August 17, 2023
Clinical Classification of Variants in the Valosin-Containing Protein Gene Associated With Multisystem Proteinopathy
Marianela Schiava, Chiseko Ikenaga, Ana Topf, et al.
Nature Medicine
|
June 1, 2021
Childhood amyotrophic lateral sclerosis caused by excess sphingolipid synthesis
Payam Mohassel, Sandra Donkervoort, Museer A Lone, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
July 1, 2025
Bi-allelic loss-of-function variants in POC5 cause a syndromic retinal, endocrine, and neuromuscular ciliopathy
Anneke T Vulto-van Silfhout, Ingrid M Jazet, Suzanne Yzer, et al.
Trials
|
May 26, 2018
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial
Rebecca A Crow, Kimberly A Hart, Michael P McDermott, et al.
Page
of 38