The New England journal of medicine, 2017-11, Vol.377 (18), p.1723-1732
- Finkel, Richard S
- Mercuri, Eugenio
- Darras, Basil T
- Connolly, Anne M
- Kuntz, Nancy L
- Kirschner, Janbernd
- Chiriboga, Claudia A
- Saito, Kayoko
- Servais, Laurent
- Tizzano, Eduardo
- Topaloglu, Haluk
- Tulinius, Már
- Montes, Jacqueline
- Glanzman, Allan M
- Bishop, Kathie
- Zhong, Z. John
- Gheuens, Sarah
- Bennett, C. Frank
- Schneider, Eugene
- Farwell, Wildon
- De Vivo, Darryl C
2017
Details
- Autor(en) / Beteiligte
- Finkel, Richard S
- Mercuri, Eugenio
- Darras, Basil T
- Connolly, Anne M
- Kuntz, Nancy L
- Kirschner, Janbernd
- Chiriboga, Claudia A
- Saito, Kayoko
- Servais, Laurent
- Tizzano, Eduardo
- Topaloglu, Haluk
- Tulinius, Már
- Montes, Jacqueline
- Glanzman, Allan M
- Bishop, Kathie
- Zhong, Z. John
- Gheuens, Sarah
- Bennett, C. Frank
- Schneider, Eugene
- Farwell, Wildon
- De Vivo, Darryl C
- Titel
- Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
- Ist Teil von
- The New England journal of medicine, 2017-11, Vol.377 (18), p.1723-1732
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2017
- Link zum Volltext
- Quelle
- MEDLINE
- Beschreibungen/Notizen
- In this phase 3 trial, among infants with spinal muscular atrophy, those who received nusinersen were more likely to achieve major motor milestones and less likely to need permanent assisted ventilation than those who underwent a sham procedure.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793, 1533-4406eISSN: 1533-4406DOI: 10.1056/NEJMoa1702752Titel-ID: cdi_swepub_primary_oai_gup_ub_gu_se_259613
- Format
- –
- Schlagworte
- Age, Age of Onset, Analysis, Antisense oligonucleotides, Babies, Care and treatment, Cell survival, Children & youth, Death, Disease-Free Survival, Double-Blind Method, Double-blind studies, Drug dosages, Drug therapy, Female, General & Internal Medicine, Hereditary diseases, Human health sciences, Humans, Infant, Infants, Injections, Spinal, Male, Mechanical ventilation, Motor ability, Motor Skills, Motor task performance, mRNA, Mutation, Neurologie, Neurology, Neuromuscular diseases, Neurons, Oligonucleotides - adverse effects, Oligonucleotides - therapeutic use, Oligonucleotides, Antisense - adverse effects, Oligonucleotides, Antisense - therapeutic use, Patient outcomes, Pediatrics, Pediatrik, Proteins, Pédiatrie, Respiration, Artificial, RNA, Messenger - drug effects, RNA, Messenger - metabolism, Sciences de la santé humaine, SMN protein, Spinal Muscular Atrophies of Childhood - drug therapy, Spinal Muscular Atrophies of Childhood - genetics, Spinal Muscular Atrophies of Childhood - mortality, Spinal Muscular Atrophies of Childhood - physiopathology, Spinal Muscular Atrophies of Childhood/drug therapy/genetics/mortality/physiopathology, Spinal muscular atrophy, Splicing, Surgery, Survival Analysis, Survival of Motor Neuron 2 Protein - genetics, Survival of Motor Neuron 2 Protein - metabolism, Survival of Motor Neuron 2 Protein/genetics/metabolism, trials