Search research articles
Contact Us
Filters
Showing results (291-300 of 303) with videos related to
Page
of 31
Sort By:
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 3
Federica Trucco, Deborah Ridout, Harriet Weststrate, et al.
Annals of Neurology
|
September 14, 2020
Clinical Variability in Spinal Muscular Atrophy Type III
Giorgia Coratti, Sonia Messina, Simona Lucibello, et al.
Annals of Neurology
|
November 18, 2017
Natural history of infantile-onset spinal muscular atrophy
Stephen 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 study
Stephen 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 study
Giorgia Coratti, Francesca Bovis, Valentina Franchino, et al.
Contemporary Clinical Trials
|
April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
Michela 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 trial
Rebecca 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 disability
Sonja 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 Trial
Michela 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 myopathy
Johann Böhm, Valérie Biancalana, Elizabeth T Dechene, et al.
Page
of 31
Search research articles
Search
Showing results (291-300 of 303) with videos related to
Sort By:
Page
of 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 3
Federica Trucco, Deborah Ridout, Harriet Weststrate, et al.
Annals of Neurology
|
September 14, 2020
Clinical Variability in Spinal Muscular Atrophy Type III
Giorgia Coratti, Sonia Messina, Simona Lucibello, et al.
Annals of Neurology
|
November 18, 2017
Natural history of infantile-onset spinal muscular atrophy
Stephen 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 study
Stephen 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 study
Giorgia Coratti, Francesca Bovis, Valentina Franchino, et al.
Contemporary Clinical Trials
|
April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
Michela 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 trial
Rebecca 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 disability
Sonja 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 Trial
Michela 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 myopathy
Johann Böhm, Valérie Biancalana, Elizabeth T Dechene, et al.
Page
of 31