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Nusinersen versus S...
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
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Mercuri, E. (författare)
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Darras, B. T. (författare)
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Chiriboga, C. A. (författare)
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visa fler...
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Day, J. W. (författare)
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Campbell, C. (författare)
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Connolly, A. M. (författare)
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Iannaccone, S. T. (författare)
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Kirschner, J. (författare)
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Kuntz, N. L. (författare)
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Saito, K. (författare)
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Shieh, P. B. (författare)
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- Tulinius, Mar, 1953 (författare)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics
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Mazzone, E. S. (författare)
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Montes, J. (författare)
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Bishop, K. M. (författare)
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Yang, Q. (författare)
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Foster, R. (författare)
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Gheuens, S. (författare)
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Bennett, C. F. (författare)
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Farwell, W. (författare)
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Schneider, E. (författare)
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De Vivo, D. C. (författare)
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Finkel, R. S. (författare)
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Cherish Study Grp, Cherish Study Grp (författare)
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(creator_code:org_t)
- 2018
- 2018
- Engelska.
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Ingår i: New England Journal of Medicine. - 0028-4793. ; 378:7, s. 625-635
- Relaterad länk:
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https://gup.ub.gu.se...
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https://doi.org/10.1...
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Abstract
Ämnesord
Stäng
- BACKGROUND Nusinersen is an antisense oligonucleotide drug that modulates pre-messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA). METHODS We conducted a multicenter, double-blind, sham-controlled, phase 3 trial of nusinersen in 126 children with SMA who had symptom onset after 6 months of age. The children were randomly assigned, in a 2: 1 ratio, to undergo intrathecal administration of nusinersen at a dose of 12 mg (nusinersen group) or a sham procedure (control group) on days 1, 29, 85, and 274. The primary end point was the least-squares mean change from baseline in the Hammersmith Functional Motor Scale-Expanded (HFMSE) score at 15 months of treatment; HFMSE scores range from 0 to 66, with higher scores indicating better motor function. Secondary end points included the percentage of children with a clinically meaningful increase from baseline in the HFMSE score (>= 3 points), an outcome that indicates improvement in at least two motor skills. RESULTS In the prespecified interim analysis, there was a least-squares mean increase from baseline to month 15 in the HFMSE score in the nusinersen group (by 4.0 points) and a least-squares mean decrease in the control group (by -1.9 points), with a significant between-group difference favoring nusinersen (least-squares mean difference in change, 5.9 points; 95% confidence interval, 3.7 to 8.1; P< 0.001). This result prompted early termination of the trial. Results of the final analysis were consistent with results of the interim analysis. In the final analysis, 57% of the children in the nusinersen group as compared with 26% in the control group had an increase from baseline to month 15 in the HFMSE score of at least 3 points (P< 0.001), and the overall incidence of adverse events was similar in the nusinersen group and the control group (93% and 100%, respectively). CONCLUSIONS Among children with later-onset SMA, those who received nusinersen had significant and clinically meaningful improvement in motor function as compared with those in the control group. (Funded by Biogen and Ionis Pharmaceuticals; CHERISH ClinicalTrials. gov number, NCT02292537.)
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Pediatrik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Pediatrics (hsv//eng)
Nyckelord
- functional motor scale
- lumbar puncture
- headache
- children
- severity
- backache
- protein
- trials
- General & Internal Medicine
Publikations- och innehållstyp
- ref (ämneskategori)
- art (ämneskategori)
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Till lärosätets databas
- Av författaren/redakt...
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Mercuri, E.
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Darras, B. T.
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Chiriboga, C. A.
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Day, J. W.
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Campbell, C.
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Connolly, A. M.
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visa fler...
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Iannaccone, S. T ...
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Kirschner, J.
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Kuntz, N. L.
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Saito, K.
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Shieh, P. B.
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Tulinius, Mar, 1 ...
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Mazzone, E. S.
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Montes, J.
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Bishop, K. M.
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Yang, Q.
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Foster, R.
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Gheuens, S.
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Bennett, C. F.
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Farwell, W.
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Schneider, E.
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De Vivo, D. C.
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Finkel, R. S.
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Cherish Study Gr ...
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visa färre...
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och Klinisk medicin
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och Pediatrik
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New England Jour ...
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Göteborgs universitet