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Basil T Darras

Showing results (181-190 of 222) with videos related to

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The Lancet. Neurology|March 20, 2021
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trialJohn W Day, Richard S Finkel, Claudia A Chiriboga, et al.
BMC Neurology|February 25, 2017
Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophyMaria C Pera, Giorgia Coratti, Nicola Forcina, et al.
Journal of Neuromuscular Diseases|February 20, 2025
Characterization of swallowing biomechanics and function in untreated infants with spinal muscular atrophy: A natural history datasetKatlyn Elizabeth McGrattan, Robert J Graham, Alicia Hofelich Mohr, et al.
Plos One|June 27, 2018
Ambulatory function in spinal muscular atrophy: Age-related patterns of progressionJacqueline Montes, Michael P McDermott, Elizabeth Mirek, et al.
Neuromuscular Disorders : NMD|January 19, 2016
Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trialsEugenio Mercuri, Richard Finkel, Jacqueline Montes, et al.
Muscle & Nerve|March 2, 2016
Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-upAnne M Connolly, Julaine M Florence, Craig M Zaidman, et al.
Contemporary Clinical Trials Communications|August 11, 2018
Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!Amy Bartlett, Stephen J Kolb, Allison Kingsley, et al.
Expert Review of Neurotherapeutics|November 13, 2025
A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophyThomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
The Lancet. Neurology|August 16, 2025
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trialThomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
Annals of Clinical and Translational Neurology|June 23, 2022
Diagnostic capabilities of nanopore long-read sequencing in muscular dystrophyChristine C Bruels, Hannah R Littel, Audrey L Daugherty, et al.
Pageof 23

Showing results (181-190 of 222) with videos related to

Sort By:
Pageof 23
The Lancet. Neurology|March 20, 2021
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trialJohn W Day, Richard S Finkel, Claudia A Chiriboga, et al.
BMC Neurology|February 25, 2017
Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophyMaria C Pera, Giorgia Coratti, Nicola Forcina, et al.
Journal of Neuromuscular Diseases|February 20, 2025
Characterization of swallowing biomechanics and function in untreated infants with spinal muscular atrophy: A natural history datasetKatlyn Elizabeth McGrattan, Robert J Graham, Alicia Hofelich Mohr, et al.
Plos One|June 27, 2018
Ambulatory function in spinal muscular atrophy: Age-related patterns of progressionJacqueline Montes, Michael P McDermott, Elizabeth Mirek, et al.
Neuromuscular Disorders : NMD|January 19, 2016
Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trialsEugenio Mercuri, Richard Finkel, Jacqueline Montes, et al.
Muscle & Nerve|March 2, 2016
Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-upAnne M Connolly, Julaine M Florence, Craig M Zaidman, et al.
Contemporary Clinical Trials Communications|August 11, 2018
Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!Amy Bartlett, Stephen J Kolb, Allison Kingsley, et al.
Expert Review of Neurotherapeutics|November 13, 2025
A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophyThomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
The Lancet. Neurology|August 16, 2025
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trialThomas O Crawford, Laurent Servais, Eugenio Mercuri, et al.
Annals of Clinical and Translational Neurology|June 23, 2022
Diagnostic capabilities of nanopore long-read sequencing in muscular dystrophyChristine C Bruels, Hannah R Littel, Audrey L Daugherty, et al.
Pageof 23