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The New England journal of medicine, ISSN 1533-4406, 11/2017, Volume 377, Issue 18, pp. 1713 - 1722
Journal Article
Journal Article
European journal of human genetics : EJHG, ISSN 1476-5438, 08/2011, Volume 20, Issue 1, pp. 27 - 32
Journal Article
The New England journal of medicine, ISSN 1533-4406, 11/2017, Volume 377, Issue 18, pp. 1723 - 1732
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... 
Medicine, General & Internal | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | Injections, Spinal | Spinal Muscular Atrophies of Childhood - genetics | Double-Blind Method | Oligonucleotides - therapeutic use | Humans | Spinal Muscular Atrophies of Childhood - drug therapy | Infant | Male | Oligonucleotides - adverse effects | Oligonucleotides, Antisense - adverse effects | RNA, Messenger - metabolism | Survival of Motor Neuron 2 Protein - metabolism | Respiration, Artificial | Disease-Free Survival | Oligonucleotides, Antisense - therapeutic use | Survival of Motor Neuron 2 Protein - genetics | Age of Onset | Survival Analysis | Female | Spinal Muscular Atrophies of Childhood - mortality | Spinal Muscular Atrophies of Childhood - physiopathology | Motor Skills | RNA, Messenger - drug effects | Care and treatment | Usage | Surgery | Patient outcomes | Motor ability | Analysis | Spinal muscular atrophy | Pediatrics | Neuromuscular diseases | Cell survival | Splicing | Neurons | SMN protein | Antisense oligonucleotides | mRNA | Infants | Motor task performance | Hereditary diseases | Children & youth | Babies | Proteins | Neurology | Ventilation | Death | Mutation | Drug therapy | Drug dosages | Age | Index Medicus | Abridged Index Medicus | trials | Pediatrik
Journal Article
Spine (Philadelphia, Pa. 1976), ISSN 0362-2436, 06/2015, Volume 40, Issue 12, pp. E675 - E693
Journal Article