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Orphanet Journal of Rare Diseases
|
October 6, 2018
The Italian neuromuscular registry: a coordinated platform where patient organizations and clinicians collaborate for data collection and multiple usage
Anna Ambrosini, Daniela Calabrese, Francesco Maria Avato, et al.
Children (Basel, Switzerland)
|
August 28, 2025
Early Predictors of Outcome in Pediatric Acquired Demyelinating Syndromes: A Retrospective Study Stratified by Final Diagnosis
Emanuela Claudia Turco, Martina Gnazzo, Sara Giordani, et al.
Muscles (Basel, Switzerland)
|
December 24, 2025
The Psychological Burden of Neuromuscular Diseases: A Narrative Review of Anxiety, Depression, Coping, and Quality of Life
Valentina Baldini, Giorgia Varallo, Andi Nuredini, et al.
Orphanet Journal of Rare Diseases
|
October 14, 2021
Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis
Giorgia Coratti, Costanza Cutrona, Maria Carmela Pera, et al.
Plos One
|
May 5, 2022
Predictive models in SMA II natural history trajectories using machine learning: A proof of concept study
Giorgia Coratti, Jacopo Lenkowicz, Stefano Patarnello, et al.
Muscle & Nerve
|
January 20, 2025
Pregnancy in the Era of Disease-Modifying Therapies for Spinal Muscular Atrophy: Considerations From a Case Report
Gabriele Vadi, Sara Loprieno, Francesca Torri, et al.
Molecular Neurobiology
|
November 26, 2024
High Expression of SMN circ4-2b-3 in SMA I Children Treated with Nusinersen is Associated with Improved Motor Outcomes
Marika Guerra, Alberto Marini, Vittoria Pagliarini, et al.
Neuromuscular Disorders : NMD
|
September 28, 2019
Development of an academic disease registry for spinal muscular atrophy
Eugenio Mercuri, Richard Finkel, MariaCristina Scoto, et al.
Journal of Clinical Medicine
|
July 14, 2023
Caregivers' Expectations on Possible Functional Changes following Disease-Modifying Treatment in Type II and III Spinal Muscular Atrophy: A Comparative Study
Maria Carmela Pera, Giorgia Coratti, Jacopo Casiraghi, et al.
Neuromuscular Disorders : NMD
|
December 4, 2019
A critical review of patient and parent caregiver oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in spinal muscular atrophy
Sonia Messina, Anna Lia Frongia, Laura Antonaci, et al.
Page
of 9
Search research articles
Search
Showing results (21-30 of 83) with videos related to
Sort By:
Page
of 9
Orphanet Journal of Rare Diseases
|
October 6, 2018
The Italian neuromuscular registry: a coordinated platform where patient organizations and clinicians collaborate for data collection and multiple usage
Anna Ambrosini, Daniela Calabrese, Francesco Maria Avato, et al.
Children (Basel, Switzerland)
|
August 28, 2025
Early Predictors of Outcome in Pediatric Acquired Demyelinating Syndromes: A Retrospective Study Stratified by Final Diagnosis
Emanuela Claudia Turco, Martina Gnazzo, Sara Giordani, et al.
Muscles (Basel, Switzerland)
|
December 24, 2025
The Psychological Burden of Neuromuscular Diseases: A Narrative Review of Anxiety, Depression, Coping, and Quality of Life
Valentina Baldini, Giorgia Varallo, Andi Nuredini, et al.
Orphanet Journal of Rare Diseases
|
October 14, 2021
Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis
Giorgia Coratti, Costanza Cutrona, Maria Carmela Pera, et al.
Plos One
|
May 5, 2022
Predictive models in SMA II natural history trajectories using machine learning: A proof of concept study
Giorgia Coratti, Jacopo Lenkowicz, Stefano Patarnello, et al.
Muscle & Nerve
|
January 20, 2025
Pregnancy in the Era of Disease-Modifying Therapies for Spinal Muscular Atrophy: Considerations From a Case Report
Gabriele Vadi, Sara Loprieno, Francesca Torri, et al.
Molecular Neurobiology
|
November 26, 2024
High Expression of SMN circ4-2b-3 in SMA I Children Treated with Nusinersen is Associated with Improved Motor Outcomes
Marika Guerra, Alberto Marini, Vittoria Pagliarini, et al.
Neuromuscular Disorders : NMD
|
September 28, 2019
Development of an academic disease registry for spinal muscular atrophy
Eugenio Mercuri, Richard Finkel, MariaCristina Scoto, et al.
Journal of Clinical Medicine
|
July 14, 2023
Caregivers' Expectations on Possible Functional Changes following Disease-Modifying Treatment in Type II and III Spinal Muscular Atrophy: A Comparative Study
Maria Carmela Pera, Giorgia Coratti, Jacopo Casiraghi, et al.
Neuromuscular Disorders : NMD
|
December 4, 2019
A critical review of patient and parent caregiver oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in spinal muscular atrophy
Sonia Messina, Anna Lia Frongia, Laura Antonaci, et al.
Page
of 9