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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 7, 2025
Current clinical applications of AAV-mediated gene therapy
Barry J Byrne, Kevin M Flanigan, Susan E Matesanz, et al.
Brain : a Journal of Neurology
|
November 2, 2013
SIL1 mutations and clinical spectrum in patients with Marinesco-Sjogren syndrome
Michael Krieger, Andreas Roos, Claudia Stendel, et al.
Neuromuscular Disorders : NMD
|
January 4, 2022
Revised upper limb module in type II and III spinal muscular atrophy: 24-month changes
Giorgia Coratti, Maria Carmela Pera, Jacqueline Montes, et al.
Brain : a Journal of Neurology
|
January 8, 2021
Natural history of Charcot-Marie-Tooth disease type 2A: a large international multicentre study
Menelaos Pipis, Shawna M E Feely, James M Polke, et al.
Journal of Comparative Effectiveness Research
|
August 28, 2020
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Craig Campbell, Richard J Barohn, Enrico Bertini, et al.
Pharmacological Research
|
September 17, 2018
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug
Laurie S Conklin, Jesse M Damsker, Eric P Hoffman, et al.
Neurology
|
February 13, 2020
A longitudinal study of CMT1A using Rasch analysis based CMT neuropathy and examination scores
Vera Fridman, Stefan Sillau, Gyula Acsadi, et al.
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.
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.
Page
of 29
Search research articles
Search
Showing results (271-280 of 289) with videos related to
Sort By:
Page
of 29
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 7, 2025
Current clinical applications of AAV-mediated gene therapy
Barry J Byrne, Kevin M Flanigan, Susan E Matesanz, et al.
Brain : a Journal of Neurology
|
November 2, 2013
SIL1 mutations and clinical spectrum in patients with Marinesco-Sjogren syndrome
Michael Krieger, Andreas Roos, Claudia Stendel, et al.
Neuromuscular Disorders : NMD
|
January 4, 2022
Revised upper limb module in type II and III spinal muscular atrophy: 24-month changes
Giorgia Coratti, Maria Carmela Pera, Jacqueline Montes, et al.
Brain : a Journal of Neurology
|
January 8, 2021
Natural history of Charcot-Marie-Tooth disease type 2A: a large international multicentre study
Menelaos Pipis, Shawna M E Feely, James M Polke, et al.
Journal of Comparative Effectiveness Research
|
August 28, 2020
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Craig Campbell, Richard J Barohn, Enrico Bertini, et al.
Pharmacological Research
|
September 17, 2018
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug
Laurie S Conklin, Jesse M Damsker, Eric P Hoffman, et al.
Neurology
|
February 13, 2020
A longitudinal study of CMT1A using Rasch analysis based CMT neuropathy and examination scores
Vera Fridman, Stefan Sillau, Gyula Acsadi, et al.
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.
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.
Page
of 29