Search research articles
Contact Us
Filters
Showing results (151-160 of 222) with videos related to
Page
of 23
Sort By:
Neurology
|
December 19, 2024
Exome and Genome Sequencing to Diagnose the Genetic Basis of Neonatal Hypotonia: An International Consortium Study
Sarah U Morton, Gregory Costain, Courtney E French, et al.
Neurology
|
January 13, 2025
Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study
Hugh J McMillan, Giovanni Baranello, Michelle A Farrar, et al.
Nature Medicine
|
December 8, 2025
Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial
Jennifer M Kwon, Francina Munell, Laure Le Goff, et al.
Journal of Human Genetics
|
October 7, 2016
The sensitivity of exome sequencing in identifying pathogenic mutations for LGMD in the United States
Hemakumar M Reddy, Kyung-Ah Cho, Monkol Lek, et al.
Neuromuscular Disorders : NMD
|
October 30, 2016
Developmental milestones in type I spinal muscular atrophy
Roberto De Sanctis, Giorgia Coratti, Amy Pasternak, et al.
Nature Medicine
|
January 28, 2026
Author Correction: Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial
Jennifer M Kwon, Francina Munell, Laure Le Goff, et al.
Muscle & Nerve
|
April 8, 2015
Spinal muscular atrophy functional composite score: A functional measure in spinal muscular atrophy
Jacqueline Montes, Allan M Glanzman, Elena S Mazzone, et al.
Neurology. Clinical Practice
|
September 3, 2021
Nusinersen Treatment in Adults With Spinal Muscular Atrophy
Tina Duong, Connie Wolford, Michael P McDermott, et al.
Journal of Neuromuscular Diseases
|
June 6, 2018
Evaluator Training and Reliability for SMA Global Nusinersen Trials1
Allan M Glanzman, Elena S Mazzone, Sally Dunaway Young, 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.
Page
of 23
Search research articles
Search
Showing results (151-160 of 222) with videos related to
Sort By:
Page
of 23
Neurology
|
December 19, 2024
Exome and Genome Sequencing to Diagnose the Genetic Basis of Neonatal Hypotonia: An International Consortium Study
Sarah U Morton, Gregory Costain, Courtney E French, et al.
Neurology
|
January 13, 2025
Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study
Hugh J McMillan, Giovanni Baranello, Michelle A Farrar, et al.
Nature Medicine
|
December 8, 2025
Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial
Jennifer M Kwon, Francina Munell, Laure Le Goff, et al.
Journal of Human Genetics
|
October 7, 2016
The sensitivity of exome sequencing in identifying pathogenic mutations for LGMD in the United States
Hemakumar M Reddy, Kyung-Ah Cho, Monkol Lek, et al.
Neuromuscular Disorders : NMD
|
October 30, 2016
Developmental milestones in type I spinal muscular atrophy
Roberto De Sanctis, Giorgia Coratti, Amy Pasternak, et al.
Nature Medicine
|
January 28, 2026
Author Correction: Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial
Jennifer M Kwon, Francina Munell, Laure Le Goff, et al.
Muscle & Nerve
|
April 8, 2015
Spinal muscular atrophy functional composite score: A functional measure in spinal muscular atrophy
Jacqueline Montes, Allan M Glanzman, Elena S Mazzone, et al.
Neurology. Clinical Practice
|
September 3, 2021
Nusinersen Treatment in Adults With Spinal Muscular Atrophy
Tina Duong, Connie Wolford, Michael P McDermott, et al.
Journal of Neuromuscular Diseases
|
June 6, 2018
Evaluator Training and Reliability for SMA Global Nusinersen Trials1
Allan M Glanzman, Elena S Mazzone, Sally Dunaway Young, 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.
Page
of 23