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Showing results (291-300 of 303) with videos related to

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Neurology. Clinical Practice|June 27, 2024
Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3Federica Trucco, Deborah Ridout, Harriet Weststrate, et al.
Annals of Neurology|September 14, 2020
Clinical Variability in Spinal Muscular Atrophy Type IIIGiorgia Coratti, Sonia Messina, Simona Lucibello, et al.
Annals of Neurology|November 18, 2017
Natural history of infantile-onset spinal muscular atrophyStephen J Kolb, Christopher S Coffey, Jon W Yankey, et al.
Annals of Clinical and Translational Neurology|February 23, 2016
Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker studyStephen J Kolb, Christopher S Coffey, Jon W Yankey, et al.
Journal of Neurology, Neurosurgery, and Psychiatry|January 30, 2026
Loss of ambulation in SMA III at the time of disease-modifying treatments: an international studyGiorgia Coratti, Francesca Bovis, Valentina Franchino, et al.
Contemporary Clinical Trials|April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophyMichela Guglieri, Kate Bushby, Michael P McDermott, et al.
Trials|May 26, 2018
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trialRebecca A Crow, Kimberly A Hart, Michael P McDermott, et al.
Human Mutation|April 13, 2021
Clinical, neuroimaging, and molecular spectrum of TECPR2-associated hereditary sensory and autonomic neuropathy with intellectual disabilitySonja Neuser, Barbara Brechmann, Gali Heimer, et al.
JAMA|April 5, 2022
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical TrialMichela Guglieri, Kate Bushby, Michael P McDermott, et al.
Human Mutation|March 8, 2012
Mutation spectrum in the large GTPase dynamin 2, and genotype-phenotype correlation in autosomal dominant centronuclear myopathyJohann Böhm, Valérie Biancalana, Elizabeth T Dechene, et al.
Pageof 31

Showing results (291-300 of 303) with videos related to

Sort By:
Pageof 31
Neurology. Clinical Practice|June 27, 2024
Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3Federica Trucco, Deborah Ridout, Harriet Weststrate, et al.
Annals of Neurology|September 14, 2020
Clinical Variability in Spinal Muscular Atrophy Type IIIGiorgia Coratti, Sonia Messina, Simona Lucibello, et al.
Annals of Neurology|November 18, 2017
Natural history of infantile-onset spinal muscular atrophyStephen J Kolb, Christopher S Coffey, Jon W Yankey, et al.
Annals of Clinical and Translational Neurology|February 23, 2016
Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker studyStephen J Kolb, Christopher S Coffey, Jon W Yankey, et al.
Journal of Neurology, Neurosurgery, and Psychiatry|January 30, 2026
Loss of ambulation in SMA III at the time of disease-modifying treatments: an international studyGiorgia Coratti, Francesca Bovis, Valentina Franchino, et al.
Contemporary Clinical Trials|April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophyMichela Guglieri, Kate Bushby, Michael P McDermott, et al.
Trials|May 26, 2018
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trialRebecca A Crow, Kimberly A Hart, Michael P McDermott, et al.
Human Mutation|April 13, 2021
Clinical, neuroimaging, and molecular spectrum of TECPR2-associated hereditary sensory and autonomic neuropathy with intellectual disabilitySonja Neuser, Barbara Brechmann, Gali Heimer, et al.
JAMA|April 5, 2022
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical TrialMichela Guglieri, Kate Bushby, Michael P McDermott, et al.
Human Mutation|March 8, 2012
Mutation spectrum in the large GTPase dynamin 2, and genotype-phenotype correlation in autosomal dominant centronuclear myopathyJohann Böhm, Valérie Biancalana, Elizabeth T Dechene, et al.
Pageof 31