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Pediatric Neurology
|
December 3, 2014
Gene therapy for muscular dystrophy: moving the field forward
Samiah Al-Zaidy, Louise Rodino-Klapac, Jerry R Mendell
Molecular Therapy. Methods & Clinical Development
|
September 7, 2023
Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec
Damon Asher, Daisy Dai, Alexa C Klimchak, et al.
Journal of Neuromuscular Diseases
|
September 13, 2021
Visualizing Muscle Sialic Acid Expression in the GNED207VTgGne-/- Cmah-/- Model of GNE Myopathy: A Comparison of Dietary and Gene Therapy Approaches
Kelly E Crowe, Deborah A Zygmunt, Kristin Heller, et al.
Neuroscience Letters
|
May 22, 2012
Gene therapy for muscular dystrophy: lessons learned and path forward
Jerry R Mendell, Louise Rodino-Klapac, Zarife Sahenk, et al.
Neurology. Clinical Practice
|
August 4, 2025
Limb-Girdle Muscular Dystrophy Scientific Workshop: A Multistakeholder Discussion Focused on Charting the Path Forward for Drug Development
Matthew P Wicklund, Lindsay N Alfano, Nicholas E Johnson, et al.
Human Gene Therapy
|
November 6, 2025
Pharmacokinetic and Pharmacodynamic Evaluation of Bidridistrogene Xeboparvovec in an Aged Murine Model of Limb-Girdle Muscular Dystrophy Type 2E/R4
Stephen Baine, Jared Cui, Young-Eun Seo, et al.
The New England Journal of Medicine
|
October 8, 2010
Dystrophin immunity in Duchenne's muscular dystrophy
Jerry R Mendell, Katherine Campbell, Louise Rodino-Klapac, et al.
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of 1
Search research articles
Search
Showing results (1-10 of 7) with videos related to
Sort By:
Page
of 1
Pediatric Neurology
|
December 3, 2014
Gene therapy for muscular dystrophy: moving the field forward
Samiah Al-Zaidy, Louise Rodino-Klapac, Jerry R Mendell
Molecular Therapy. Methods & Clinical Development
|
September 7, 2023
Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec
Damon Asher, Daisy Dai, Alexa C Klimchak, et al.
Journal of Neuromuscular Diseases
|
September 13, 2021
Visualizing Muscle Sialic Acid Expression in the GNED207VTgGne-/- Cmah-/- Model of GNE Myopathy: A Comparison of Dietary and Gene Therapy Approaches
Kelly E Crowe, Deborah A Zygmunt, Kristin Heller, et al.
Neuroscience Letters
|
May 22, 2012
Gene therapy for muscular dystrophy: lessons learned and path forward
Jerry R Mendell, Louise Rodino-Klapac, Zarife Sahenk, et al.
Neurology. Clinical Practice
|
August 4, 2025
Limb-Girdle Muscular Dystrophy Scientific Workshop: A Multistakeholder Discussion Focused on Charting the Path Forward for Drug Development
Matthew P Wicklund, Lindsay N Alfano, Nicholas E Johnson, et al.
Human Gene Therapy
|
November 6, 2025
Pharmacokinetic and Pharmacodynamic Evaluation of Bidridistrogene Xeboparvovec in an Aged Murine Model of Limb-Girdle Muscular Dystrophy Type 2E/R4
Stephen Baine, Jared Cui, Young-Eun Seo, et al.
The New England Journal of Medicine
|
October 8, 2010
Dystrophin immunity in Duchenne's muscular dystrophy
Jerry R Mendell, Katherine Campbell, Louise Rodino-Klapac, et al.
Page
of 1