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Journal of Neuromuscular Diseases|March 1, 2024
Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3Amy Wolfe, Georgia Stimpson, Danielle Ramsey, et al.
The American Journal of Bioethics : AJOB|April 28, 2009
Unintended changes in cognition, mood, and behavior arising from cell-based interventions for neurological conditions: ethical challengesP S Duggan, A W Siegel, D M Blass, et al.
Brain : a Journal of Neurology|November 2, 2013
SIL1 mutations and clinical spectrum in patients with Marinesco-Sjogren syndromeMichael Krieger, Andreas Roos, Claudia Stendel, et al.
Muscle & Nerve|July 30, 2021
Different trajectories in upper limb and gross motor function in spinal muscular atrophyGiorgia Coratti, Maria Carmela Pera, Jacqueline Montes, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 7, 2025
Current clinical applications of AAV-mediated gene therapyBarry J Byrne, Kevin M Flanigan, Susan E Matesanz, et al.
Neuromuscular Disorders : NMD|January 4, 2022
Revised upper limb module in type II and III spinal muscular atrophy: 24-month changesGiorgia Coratti, Maria Carmela Pera, Jacqueline Montes, et al.
Emerging Infectious Diseases|November 26, 2024
Bartonella quintana Infection in Kidney Transplant Recipients from Donor Experiencing Homelessness, United States, 2022Amy M Beeson, Shannan N Rich, Michael E Russo, et al.
BMC Medical Genetics|March 24, 2016
Variants in CXCR4 associate with juvenile idiopathic arthritis susceptibilityTerri H Finkel, Jin Li, Zhi Wei, et al.
Nature Communications|July 21, 2024
The AKT2/SIRT5/TFEB pathway as a potential therapeutic target in non-neovascular AMDSayan Ghosh, Ruchi Sharma, Sridhar Bammidi, et al.
Journal of Comparative Effectiveness Research|August 28, 2020
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophyCraig Campbell, Richard J Barohn, Enrico Bertini, et al.
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