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Molecular Genetics and Metabolism
|
August 5, 2025
Institutional readiness for novel therapeutics: A framework for multidisciplinary integration
Elizabeth G Ames, Nicholas A Borja, Russell J Butterfield, et al.
Annals of Clinical and Translational Neurology
|
July 15, 2025
BCS1L-Associated Disease: 5'-UTR Variant Shifts the Phenotype Towards Axonal Neuropathy
Rotem Orbach, Nunziata Maio, Russell J Butterfield, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 30, 2025
Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchenne muscular dystrophy: Initial observations from a phase 1b trial
Sarah P Sherlock, Daniel I Levy, Avery McIntosh, et al.
Muscle & Nerve
|
August 31, 2018
Modified dynamic gait index and limits of stability in myotonic dystrophy type 1
Evan M Pucillo, Melissa M Mcintyre, Mary Pautler, et al.
Plos One
|
August 23, 2022
Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial
Francesco Muntoni, Michela Guglieri, Jean K Mah, et al.
Nature Medicine
|
June 27, 2025
AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial
Russell J Butterfield, Perry B Shieh, Huihua Li, et al.
International Journal of Neonatal Screening
|
April 23, 2024
Long-Term Follow-Up Cares and Check Initiative: A Program to Advance Long-Term Follow-Up in Newborns Identified with a Disease through Newborn Screening
Mei Lietsch, Kee Chan, Jennifer Taylor, et al.
Human Mutation
|
September 17, 2013
Position of glycine substitutions in the triple helix of COL6A1, COL6A2, and COL6A3 is correlated with severity and mode of inheritance in collagen VI myopathies
Russell J Butterfield, A Reghan Foley, Jahannaz Dastgir, et al.
The Journal of Physiology
|
August 31, 2024
Reductions in functional muscle mass and ability to ambulate in Duchenne muscular dystrophy from ages 4 to 24 years
William J Evans, Marc Hellerstein, Russell J Butterfield, et al.
Muscle & Nerve
|
April 25, 2020
Spinal muscular atrophy care in the COVID-19 pandemic era
Aravindhan Veerapandiyan, Anne M Connolly, Richard S Finkel, et al.
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Search research articles
Search
Showing results (31-40 of 73) with videos related to
Sort By:
Page
of 8
Molecular Genetics and Metabolism
|
August 5, 2025
Institutional readiness for novel therapeutics: A framework for multidisciplinary integration
Elizabeth G Ames, Nicholas A Borja, Russell J Butterfield, et al.
Annals of Clinical and Translational Neurology
|
July 15, 2025
BCS1L-Associated Disease: 5'-UTR Variant Shifts the Phenotype Towards Axonal Neuropathy
Rotem Orbach, Nunziata Maio, Russell J Butterfield, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 30, 2025
Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchenne muscular dystrophy: Initial observations from a phase 1b trial
Sarah P Sherlock, Daniel I Levy, Avery McIntosh, et al.
Muscle & Nerve
|
August 31, 2018
Modified dynamic gait index and limits of stability in myotonic dystrophy type 1
Evan M Pucillo, Melissa M Mcintyre, Mary Pautler, et al.
Plos One
|
August 23, 2022
Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial
Francesco Muntoni, Michela Guglieri, Jean K Mah, et al.
Nature Medicine
|
June 27, 2025
AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial
Russell J Butterfield, Perry B Shieh, Huihua Li, et al.
International Journal of Neonatal Screening
|
April 23, 2024
Long-Term Follow-Up Cares and Check Initiative: A Program to Advance Long-Term Follow-Up in Newborns Identified with a Disease through Newborn Screening
Mei Lietsch, Kee Chan, Jennifer Taylor, et al.
Human Mutation
|
September 17, 2013
Position of glycine substitutions in the triple helix of COL6A1, COL6A2, and COL6A3 is correlated with severity and mode of inheritance in collagen VI myopathies
Russell J Butterfield, A Reghan Foley, Jahannaz Dastgir, et al.
The Journal of Physiology
|
August 31, 2024
Reductions in functional muscle mass and ability to ambulate in Duchenne muscular dystrophy from ages 4 to 24 years
William J Evans, Marc Hellerstein, Russell J Butterfield, et al.
Muscle & Nerve
|
April 25, 2020
Spinal muscular atrophy care in the COVID-19 pandemic era
Aravindhan Veerapandiyan, Anne M Connolly, Richard S Finkel, et al.
Page
of 8