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Journal of Pediatric Rehabilitation Medicine
|
July 19, 2021
Psychometric properties of the PEDI-CAT for children and youth with spinal muscular atrophy
Maria Fragala-Pinkham, Amy Pasternak, Michael P McDermott, et al.
Annals of Clinical and Translational Neurology
|
May 30, 2019
Neurofilament as a potential biomarker for spinal muscular atrophy
Basil T Darras, Thomas O Crawford, Richard S Finkel, et al.
Neurology
|
June 27, 2025
Spinal Muscular Atrophy Functional Composite Score Revised (SMA-FCR) in Untreated and Nusinersen-Treated Patient Cohorts
Amy Pasternak, Michael P McDermott, Jacqueline Montes, et al.
Neurology
|
February 8, 2024
Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study
Thomas O Crawford, Basil T Darras, John W Day, et al.
Pediatric Physical Therapy : the Official Publication of the Section on Pediatrics of the American Physical Therapy Association
|
November 18, 2011
Validation of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)
Allan M Glanzman, Michael P McDermott, Jacqueline Montes, et al.
Neurology
|
April 26, 2019
Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies
Basil T Darras, Claudia A Chiriboga, Susan T Iannaccone, et al.
JAMA Neurology
|
March 7, 2022
Multicenter Consensus Approach to Evaluation of Neonatal Hypotonia in the Genomic Era: A Review
Sarah U Morton, John Christodoulou, Gregory Costain, et al.
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.
Page
of 31
Search research articles
Search
Showing results (221-230 of 303) with videos related to
Sort By:
Page
of 31
Journal of Pediatric Rehabilitation Medicine
|
July 19, 2021
Psychometric properties of the PEDI-CAT for children and youth with spinal muscular atrophy
Maria Fragala-Pinkham, Amy Pasternak, Michael P McDermott, et al.
Annals of Clinical and Translational Neurology
|
May 30, 2019
Neurofilament as a potential biomarker for spinal muscular atrophy
Basil T Darras, Thomas O Crawford, Richard S Finkel, et al.
Neurology
|
June 27, 2025
Spinal Muscular Atrophy Functional Composite Score Revised (SMA-FCR) in Untreated and Nusinersen-Treated Patient Cohorts
Amy Pasternak, Michael P McDermott, Jacqueline Montes, et al.
Neurology
|
February 8, 2024
Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study
Thomas O Crawford, Basil T Darras, John W Day, et al.
Pediatric Physical Therapy : the Official Publication of the Section on Pediatrics of the American Physical Therapy Association
|
November 18, 2011
Validation of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)
Allan M Glanzman, Michael P McDermott, Jacqueline Montes, et al.
Neurology
|
April 26, 2019
Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies
Basil T Darras, Claudia A Chiriboga, Susan T Iannaccone, et al.
JAMA Neurology
|
March 7, 2022
Multicenter Consensus Approach to Evaluation of Neonatal Hypotonia in the Genomic Era: A Review
Sarah U Morton, John Christodoulou, Gregory Costain, et al.
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.
Page
of 31