The New England journal of medicine, 2018-02, Vol.378 (7), p.625-635
- Mercuri, Eugenio
- Darras, Basil T
- Chiriboga, Claudia A
- Day, John W
- Campbell, Craig
- Connolly, Anne M
- Iannaccone, Susan T
- Kirschner, Janbernd
- Kuntz, Nancy L
- Saito, Kayoko
- Shieh, Perry B
- Tulinius, Már
- Mazzone, Elena S
- Montes, Jacqueline
- Bishop, Kathie M
- Yang, Qingqing
- Foster, Richard
- Gheuens, Sarah
- Bennett, C. Frank
- Farwell, Wildon
- Schneider, Eugene
- De Vivo, Darryl C
- Finkel, Richard S
2018
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Details
- Autor(en) / Beteiligte
- Mercuri, Eugenio
- Darras, Basil T
- Chiriboga, Claudia A
- Day, John W
- Campbell, Craig
- Connolly, Anne M
- Iannaccone, Susan T
- Kirschner, Janbernd
- Kuntz, Nancy L
- Saito, Kayoko
- Shieh, Perry B
- Tulinius, Már
- Mazzone, Elena S
- Montes, Jacqueline
- Bishop, Kathie M
- Yang, Qingqing
- Foster, Richard
- Gheuens, Sarah
- Bennett, C. Frank
- Farwell, Wildon
- Schneider, Eugene
- De Vivo, Darryl C
- Finkel, Richard S
- Titel
- Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
- Ist Teil von
- The New England journal of medicine, 2018-02, Vol.378 (7), p.625-635
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2018
- Quelle
- MEDLINE
- Beschreibungen/Notizen
- In this phase 3 trial, among children with later-onset spinal muscular atrophy, those who received nusinersen had improvement in motor-function scores and those who underwent a sham procedure did not.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793, 1533-4406eISSN: 1533-4406DOI: 10.1056/NEJMoa1710504Titel-ID: cdi_swepub_primary_oai_gup_ub_gu_se_265106
- Format
- –
- Schlagworte
- Age of Onset, Antisense oligonucleotides, backache, Child, Child, Preschool, Children, Children & youth, Clinical trials, Double-Blind Method, Drug dosages, Female, functional motor scale, General & Internal Medicine, headache, Human health sciences, Humans, Infant, Injections, Spinal, Least-Squares Analysis, Life Sciences, lumbar puncture, Male, Motor skill, Motor Skills, mRNA, Mutation, Neurologie, Neurology, Neuromuscular diseases, Oligonucleotides - adverse effects, Oligonucleotides - therapeutic use, Oligonucleotides, Antisense - adverse effects, Oligonucleotides, Antisense - therapeutic use, Oligonucleotides, Antisense/adverse effects/therapeutic use, Oligonucleotides/adverse effects/therapeutic use, Pediatrics, Pediatrik, protein, Proteins, Pédiatrie, Sciences de la santé humaine, severity, SMN protein, Spinal Muscular Atrophies of Childhood - drug therapy, Spinal Muscular Atrophies of Childhood - physiopathology, Spinal Muscular Atrophies of Childhood/drug therapy/physiopathology, Spinal muscular atrophy, Splicing, trials