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T Darras

Showing results (171-180 of 303) with videos related to

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Journal of Comparative Effectiveness Research|October 25, 2021
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophyPerry B Shieh, Gary Elfring, Panayiota Trifillis, et al.
Neuromuscular Disorders : NMD|April 28, 2018
Comprehensive nutritional and metabolic assessment in patients with spinal muscular atrophy: Opportunity for an individualized approachEnid E Martinez, Nicolle Quinn, Kayla Arouchon, et al.
Neuromuscular Disorders : NMD|January 22, 2002
Primary gamma-sarcoglycanopathy (LGMD 2C): broadening of the mutational spectrum guided by the immunohistochemical profileC G Bönnemann, J Wong, K J Jones, et al.
Journal of Neuromuscular Diseases|February 22, 2020
Scoliosis Surgery Significantly Impacts Motor Abilities in Higher-functioning Individuals with Spinal Muscular Atrophy1Sally Dunaway Young, Jacqueline Montes, Rachel Salazar, et al.
Muscle & Nerve|April 6, 2022
Distribution of weight, stature, and growth status in children and adolescents with spinal muscular atrophy: An observational retrospective study in the United StatesBasil T Darras, Sabrina Guye, Janine Hoffart, et al.
Plos One|March 15, 2019
Systemic nature of spinal muscular atrophy revealed by studying insurance claimsScott L Lipnick, Denis M Agniel, Rahul Aggarwal, et al.
Annals of Neurology|February 28, 2017
Quantitative muscle ultrasound detects disease progression in Duchenne muscular dystrophyCraig M Zaidman, Jim S Wu, Kush Kapur, et al.
Journal of Neuromuscular Diseases|September 4, 2023
Erratum to: Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec TreatmentKatlyn E McGrattan, Richard D Shell, Rebecca Hurst-Davis, et al.
Journal of Neuromuscular Diseases|April 24, 2023
Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec TreatmentKatlyn E McGrattan, Richard D Shell, Rebecca Hurst-Davis, et al.
American Journal of Human Genetics|March 1, 1987
Congenital adrenal hypoplasia, myopathy, and glycerol kinase deficiency: molecular genetic evidence for deletionsU Francke, J F Harper, B T Darras, et al.
Pageof 31

Showing results (171-180 of 303) with videos related to

Sort By:
Pageof 31
Journal of Comparative Effectiveness Research|October 25, 2021
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophyPerry B Shieh, Gary Elfring, Panayiota Trifillis, et al.
Neuromuscular Disorders : NMD|April 28, 2018
Comprehensive nutritional and metabolic assessment in patients with spinal muscular atrophy: Opportunity for an individualized approachEnid E Martinez, Nicolle Quinn, Kayla Arouchon, et al.
Neuromuscular Disorders : NMD|January 22, 2002
Primary gamma-sarcoglycanopathy (LGMD 2C): broadening of the mutational spectrum guided by the immunohistochemical profileC G Bönnemann, J Wong, K J Jones, et al.
Journal of Neuromuscular Diseases|February 22, 2020
Scoliosis Surgery Significantly Impacts Motor Abilities in Higher-functioning Individuals with Spinal Muscular Atrophy1Sally Dunaway Young, Jacqueline Montes, Rachel Salazar, et al.
Muscle & Nerve|April 6, 2022
Distribution of weight, stature, and growth status in children and adolescents with spinal muscular atrophy: An observational retrospective study in the United StatesBasil T Darras, Sabrina Guye, Janine Hoffart, et al.
Plos One|March 15, 2019
Systemic nature of spinal muscular atrophy revealed by studying insurance claimsScott L Lipnick, Denis M Agniel, Rahul Aggarwal, et al.
Annals of Neurology|February 28, 2017
Quantitative muscle ultrasound detects disease progression in Duchenne muscular dystrophyCraig M Zaidman, Jim S Wu, Kush Kapur, et al.
Journal of Neuromuscular Diseases|September 4, 2023
Erratum to: Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec TreatmentKatlyn E McGrattan, Richard D Shell, Rebecca Hurst-Davis, et al.
Journal of Neuromuscular Diseases|April 24, 2023
Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec TreatmentKatlyn E McGrattan, Richard D Shell, Rebecca Hurst-Davis, et al.
American Journal of Human Genetics|March 1, 1987
Congenital adrenal hypoplasia, myopathy, and glycerol kinase deficiency: molecular genetic evidence for deletionsU Francke, J F Harper, B T Darras, et al.
Pageof 31