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Neurology
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May 11, 2022
Randomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
Florian P Thomas, Thomas H Brannagan, Russell J Butterfield, et al.
Plos One
|
December 11, 2025
Establishing biomarkers and clinical endpoints in myotonic dystrophy type 1 (END-DM1): Protocol of an international natural history study
Karlien Mul, Kate Eichinger, Man Hung, et al.
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics
|
February 24, 2021
Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study
Stacy A Rudnicki, Jinsy A Andrews, Tina Duong, et al.
Neurology. Genetics
|
April 6, 2026
Quantitative Measurement of Glycosylated ⍺-Dystroglycan as a Biomarker for Disease Severity in Limb-Girdle Muscular Dystrophy Type 2I/R9
John Vissing, Tahseen Mozaffar, Nicholas E Johnson, et al.
Journal of Clinical Medicine
|
September 28, 2024
Association between Reported Sleep Disorders and Behavioral Issues in Children with Myotonic Dystrophy Type 1-Results from a Retrospective Analysis in Italy
Federica Trucco, Andrea Lizio, Elisabetta Roma, et al.
The Lancet. Neurology
|
February 21, 2023
Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
Charles A Thornton, Richard Thomas Moxley, Katy Eichinger, et al.
Annals of Clinical and Translational Neurology
|
April 16, 2025
Clinical Trial Readiness in Limb Girdle Muscular Dystrophy R1 (LGMDR1): A GRASP Consortium Study
Stephanie M Hunn, Lindsay N Alfano, Aileen Jones, et al.
Muscle & Nerve
|
April 16, 2022
Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy
Jeffrey M Statland, Craig Campbell, Urvi Desai, et al.
Neurology
|
February 26, 2026
Prospective Study of Video Hand Opening Time as a Quantitative Measurement of Myotonia in Patients With Myotonic Dystrophy Type 1
Kristofoor E Leeuwenberg, Valeria A Sansone, Johanna Hamel, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 7, 2025
Current clinical applications of AAV-mediated gene therapy
Barry J Byrne, Kevin M Flanigan, Susan E Matesanz, et al.
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Search research articles
Search
Showing results (81-90 of 95) with videos related to
Sort By:
Page
of 10
Neurology
|
May 11, 2022
Randomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
Florian P Thomas, Thomas H Brannagan, Russell J Butterfield, et al.
Plos One
|
December 11, 2025
Establishing biomarkers and clinical endpoints in myotonic dystrophy type 1 (END-DM1): Protocol of an international natural history study
Karlien Mul, Kate Eichinger, Man Hung, et al.
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics
|
February 24, 2021
Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study
Stacy A Rudnicki, Jinsy A Andrews, Tina Duong, et al.
Neurology. Genetics
|
April 6, 2026
Quantitative Measurement of Glycosylated ⍺-Dystroglycan as a Biomarker for Disease Severity in Limb-Girdle Muscular Dystrophy Type 2I/R9
John Vissing, Tahseen Mozaffar, Nicholas E Johnson, et al.
Journal of Clinical Medicine
|
September 28, 2024
Association between Reported Sleep Disorders and Behavioral Issues in Children with Myotonic Dystrophy Type 1-Results from a Retrospective Analysis in Italy
Federica Trucco, Andrea Lizio, Elisabetta Roma, et al.
The Lancet. Neurology
|
February 21, 2023
Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
Charles A Thornton, Richard Thomas Moxley, Katy Eichinger, et al.
Annals of Clinical and Translational Neurology
|
April 16, 2025
Clinical Trial Readiness in Limb Girdle Muscular Dystrophy R1 (LGMDR1): A GRASP Consortium Study
Stephanie M Hunn, Lindsay N Alfano, Aileen Jones, et al.
Muscle & Nerve
|
April 16, 2022
Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy
Jeffrey M Statland, Craig Campbell, Urvi Desai, et al.
Neurology
|
February 26, 2026
Prospective Study of Video Hand Opening Time as a Quantitative Measurement of Myotonia in Patients With Myotonic Dystrophy Type 1
Kristofoor E Leeuwenberg, Valeria A Sansone, Johanna Hamel, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 7, 2025
Current clinical applications of AAV-mediated gene therapy
Barry J Byrne, Kevin M Flanigan, Susan E Matesanz, et al.
Page
of 10