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Pediatrics
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August 27, 2020
Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes
Megan A Waldrop, Cassandra Karingada, Mike A Storey, et al.
Journal of Neuromuscular Diseases
|
November 19, 2023
Findings from the Longitudinal CINRG Becker Natural History Study
Paula R Clemens, Heather Gordish-Dressman, Gabriela Niizawa, et al.
Journal of Neuromuscular Diseases
|
March 13, 2023
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)
Richard S Finkel, Basil T Darras, Jerry R Mendell, et al.
Plos One
|
October 19, 2020
Medical management of muscle weakness in Duchenne muscular dystrophy
Sarah R Rivera, Sumit K Jhamb, Hoda Z Abdel-Hamid, et al.
Journal of Neuromuscular Diseases
|
November 19, 2023
Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in Duchenne Muscular Dystrophy (DMD): Meeting Report from the 3rd International Muscle-Bone Interactions Meeting 7th and 14th November 2022
Kim Phung, Nicola Crabtree, Anne M Connolly, et al.
Neuromuscular Disorders : NMD
|
December 24, 2023
Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio
Megan A Waldrop, Shannon Chagat, Michael Storey, et al.
Muscle & Nerve
|
February 2, 2019
Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy
Anne M Connolly, Craig M Zaidman, Paul T Golumbek, et al.
Medicine
|
July 3, 2019
Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy
Lindsay N Alfano, Jay S Charleston, Anne M Connolly, et al.
The New England Journal of Medicine
|
November 2, 2017
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Richard S Finkel, Eugenio Mercuri, Basil T Darras, et al.
Neurology
|
March 16, 2018
A multinational study on motor function in early-onset FSHD
Jean K Mah, Jia Feng, Marni B Jacobs, et al.
Page
of 12
Search research articles
Search
Showing results (81-90 of 118) with videos related to
Sort By:
Page
of 12
Pediatrics
|
August 27, 2020
Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes
Megan A Waldrop, Cassandra Karingada, Mike A Storey, et al.
Journal of Neuromuscular Diseases
|
November 19, 2023
Findings from the Longitudinal CINRG Becker Natural History Study
Paula R Clemens, Heather Gordish-Dressman, Gabriela Niizawa, et al.
Journal of Neuromuscular Diseases
|
March 13, 2023
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)
Richard S Finkel, Basil T Darras, Jerry R Mendell, et al.
Plos One
|
October 19, 2020
Medical management of muscle weakness in Duchenne muscular dystrophy
Sarah R Rivera, Sumit K Jhamb, Hoda Z Abdel-Hamid, et al.
Journal of Neuromuscular Diseases
|
November 19, 2023
Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in Duchenne Muscular Dystrophy (DMD): Meeting Report from the 3rd International Muscle-Bone Interactions Meeting 7th and 14th November 2022
Kim Phung, Nicola Crabtree, Anne M Connolly, et al.
Neuromuscular Disorders : NMD
|
December 24, 2023
Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio
Megan A Waldrop, Shannon Chagat, Michael Storey, et al.
Muscle & Nerve
|
February 2, 2019
Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy
Anne M Connolly, Craig M Zaidman, Paul T Golumbek, et al.
Medicine
|
July 3, 2019
Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy
Lindsay N Alfano, Jay S Charleston, Anne M Connolly, et al.
The New England Journal of Medicine
|
November 2, 2017
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Richard S Finkel, Eugenio Mercuri, Basil T Darras, et al.
Neurology
|
March 16, 2018
A multinational study on motor function in early-onset FSHD
Jean K Mah, Jia Feng, Marni B Jacobs, et al.
Page
of 12