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Nusinersen versus S...
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
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Finkel, R. S. (author)
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Mercuri, E. (author)
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Darras, B. T. (author)
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Connolly, A. M. (author)
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Kuntz, N. L. (author)
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Kirschner, J. (author)
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Chiriboga, C. A. (author)
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Saito, K. (author)
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Servais, L. (author)
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Tizzano, E. (author)
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Topaloglu, H. (author)
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- Tulinius, Mar, 1953 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics
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Montes, J. (author)
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Glanzman, A. M. (author)
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Bishop, K. (author)
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Zhong, Z. J. (author)
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Gheuens, S. (author)
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Bennett, C. F. (author)
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Schneider, E. (author)
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Farwell, W. (author)
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De Vivo, D. C. (author)
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Endear Study Grp, Endear Study Grp (author)
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(creator_code:org_t)
- 2017
- 2017
- English.
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In: New England Journal of Medicine. - 0028-4793. ; 377:18, s. 1723-1732
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https://gup.ub.gu.se...
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https://doi.org/10.1...
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Abstract
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- BACKGROUND & para;& para;Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide drug that modifies pre-messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein.& para;& para;METHODS & para;& para;We conducted a randomized, double-blind, sham-controlled, phase 3 efficacy and safety trial of nusinersen in infants with spinal muscular atrophy. The primary end points were a motor-milestone response (defined according to results on the Hammersmith Infant Neurological Examination) and event-free survival (time to death or the use of permanent assisted ventilation). Secondary end points included over all survival and subgroup analyses of event-free survival according to disease duration at screening. Only the first primary end point was tested in a prespecified interim analysis. To control the overall type I error rate at 0.05, a hierarchical testing strategy was used for the second primary end point and the secondary end points in the final analysis.& para;& para;RESULTS & para;& para;In the interim analysis, a significantly higher percentage of infants in the nusinersen group than in the control group had a motor-milestone response (21 of 51 infants [41 %] vs. 0 of 27 [0%], P<0.001), and this result prompted early termination of the trial. In the final analysis, a significantly higher percentage of infants in the nusinersen group than in the control group had a motor-milestone response (37 of 73 infants [51%] vs. 0 of 37 [0%]), and the likelihood of event-free survival was higher in the nusinersen group than in the control group (hazard ratio for death or the use of permanent assisted ventilation, 0.53; P=0.005). The likelihood of overall survival was higher in the nusinersen group than in the control group (hazard ratio for death, 0.37; P=0.004), and infants with a shorter disease duration at screening were more likely than those with a longer disease duration to benefit from nusinersen. The incidence and severity of adverse events were similar in the two groups.& para;& para;CONCLUSIONS & para;& para;Among infants with spinal muscular atrophy, those who received nusinersen were more likely to be alive and have improvements in motor function than those in the control group. Early treatment may be necessary to maximize the benefit of the drug.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Pediatrik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Pediatrics (hsv//eng)
Keyword
- trials
- General & Internal Medicine
Publication and Content Type
- ref (subject category)
- art (subject category)
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- By the author/editor
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Finkel, R. S.
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Mercuri, E.
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Darras, B. T.
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Connolly, A. M.
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Kuntz, N. L.
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Kirschner, J.
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show more...
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Chiriboga, C. A.
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Saito, K.
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Servais, L.
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Tizzano, E.
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Topaloglu, H.
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Tulinius, Mar, 1 ...
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Montes, J.
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Glanzman, A. M.
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Bishop, K.
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Zhong, Z. J.
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Gheuens, S.
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Bennett, C. F.
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Schneider, E.
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Farwell, W.
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De Vivo, D. C.
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Endear Study Grp ...
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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Pediatrics
- Articles in the publication
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New England Jour ...
- By the university
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University of Gothenburg