Search research articles
Contact Us
Filters
Showing results (81-90 of 173) with videos related to
Page
of 18
Sort By:
Neuromuscular Disorders : NMD
|
February 11, 2022
Corrigendum to 'Population pharmacokinetics-based recommendations for a single delayed or missed dose of nusinersen': Neuromuscular Disorders 31 (2021) 310-318/doi: 10.1016/j.nmd.2021.02.014
Drew MacCannell, Zdenek Berger, Lilly East, et al.
Brain : a Journal of Neurology
|
December 23, 2025
Treatment evolution in spinal muscular atrophy: insights from the SMArtCARE registry
Cornelia Voigt-Müller, Michelle Pfaffenlehner, Günther Bernert, et al.
Neuropediatrics
|
December 1, 2021
Clinical and Genetic Aspects of Juvenile Onset Pompe Disease
Johanna Holzwarth, Nadja Minopoli, Charlotte Pfrimmer, et al.
Der Nervenarzt
|
January 9, 2019
[Treatment evaluation in patients with 5q-associated spinal muscular atrophy : Real-world experience]
Maggie C Walter, Bianca Dräger, Rene Günther, et al.
Journal of Neuromuscular Diseases
|
April 26, 2018
Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany
Astrid Pechmann, Thorsten Langer, David Schorling, et al.
Neuromuscular Disorders : NMD
|
August 16, 2017
A comparative study of care practices for young boys with Duchenne muscular dystrophy between Japan and European countries: Implications of early diagnosis
Fumi Takeuchi, Hirofumi Komaki, Zentaro Yamagata, et al.
Frontiers in Pediatrics
|
November 30, 2023
High-sensitive cardiac troponin I (hs-cTnI) concentrations in newborns diagnosed with spinal muscular atrophy
Jessika Johannsen, Deike Weiss, Joenna Driemeyer, et al.
Journal of Neurology
|
February 9, 2017
Predictors of Health-Related Quality of Life in boys with Duchenne muscular dystrophy from six European countries
Christiane Otto, Birgit F Steffensen, Ann-Lisbeth Højberg, et al.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society
|
August 9, 2020
European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy
Janbernd Kirschner, Nina Butoianu, Nathalie Goemans, et al.
Journal of Comparative Effectiveness Research
|
August 16, 2019
Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry
Francesco Muntoni, Isabelle Desguerre, Michela Guglieri, et al.
Page
of 18
Search research articles
Search
Showing results (81-90 of 173) with videos related to
Sort By:
Page
of 18
Neuromuscular Disorders : NMD
|
February 11, 2022
Corrigendum to 'Population pharmacokinetics-based recommendations for a single delayed or missed dose of nusinersen': Neuromuscular Disorders 31 (2021) 310-318/doi: 10.1016/j.nmd.2021.02.014
Drew MacCannell, Zdenek Berger, Lilly East, et al.
Brain : a Journal of Neurology
|
December 23, 2025
Treatment evolution in spinal muscular atrophy: insights from the SMArtCARE registry
Cornelia Voigt-Müller, Michelle Pfaffenlehner, Günther Bernert, et al.
Neuropediatrics
|
December 1, 2021
Clinical and Genetic Aspects of Juvenile Onset Pompe Disease
Johanna Holzwarth, Nadja Minopoli, Charlotte Pfrimmer, et al.
Der Nervenarzt
|
January 9, 2019
[Treatment evaluation in patients with 5q-associated spinal muscular atrophy : Real-world experience]
Maggie C Walter, Bianca Dräger, Rene Günther, et al.
Journal of Neuromuscular Diseases
|
April 26, 2018
Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany
Astrid Pechmann, Thorsten Langer, David Schorling, et al.
Neuromuscular Disorders : NMD
|
August 16, 2017
A comparative study of care practices for young boys with Duchenne muscular dystrophy between Japan and European countries: Implications of early diagnosis
Fumi Takeuchi, Hirofumi Komaki, Zentaro Yamagata, et al.
Frontiers in Pediatrics
|
November 30, 2023
High-sensitive cardiac troponin I (hs-cTnI) concentrations in newborns diagnosed with spinal muscular atrophy
Jessika Johannsen, Deike Weiss, Joenna Driemeyer, et al.
Journal of Neurology
|
February 9, 2017
Predictors of Health-Related Quality of Life in boys with Duchenne muscular dystrophy from six European countries
Christiane Otto, Birgit F Steffensen, Ann-Lisbeth Højberg, et al.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society
|
August 9, 2020
European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy
Janbernd Kirschner, Nina Butoianu, Nathalie Goemans, et al.
Journal of Comparative Effectiveness Research
|
August 16, 2019
Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry
Francesco Muntoni, Isabelle Desguerre, Michela Guglieri, et al.
Page
of 18