Drug treatment for spinal muscular atrophy types II and III
Información
- DOI:
- https://doi.org/10.1002/14651858.CD006282.pub3Copiar DOI
- Base de datos:
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- Cochrane Database of Systematic Reviews
- Versión publicada:
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- 07 diciembre 2011see what's new
- Tipo:
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- Intervention
- Etapa:
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- Review
- Grupo Editorial Cochrane:
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Grupo Cochrane de Neuromuscular
- Copyright:
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- Copyright © 2011 The Cochrane Collaboration. Published by John Wiley & Sons, Ltd.
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Autores
Contributions of authors
All authors contributed substantially to the concept and design of the review. Dr Bosboom and Dr Vrancken performed data extraction and analyses for the original review. Dr Bosboom wrote the first draft of the original review and the other co‐authors contributed to subsequent revisions for important intellectual content. Dr Wadman and Dr Vrancken updated the review in 2011.
Sources of support
Internal sources
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University Medical Center Utrecht, Department of Neurology and Neuromuscular diseases, Utrecht, Netherlands.
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University Medical Center Utrecht, Department of Child Neurology, Utrecht, Netherlands.
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Texas Scottish Rite Hospital for Children, Dallas, USA.
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Cochrane Neuromuscular Disease Group, King's College London School of Medicine, London, UK.
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University Medical Center Utrecht, Department of Biostatics and Clinical Epidemiology, Utrecht, Netherlands.
External sources
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No sources of support supplied
Declarations of interest
Dr Iannaccone was involved in the trial of riluzole as one of the investigators and authors (Russman 2003). She was involved in a trial of the efficacy of creatine for children with SMA types II and III as investigator and author (Wong 2007) and she was involved in a trial of the efficacy of riluzole (not published). She has a contract with ISIS for a trial of 396443‐CS1 in SMA patients. She also receives funding for research from PTC Therapeutics and GSK for studies in Duchenne muscular dystrophy.
Drs Van den Berg, Vrancken and Wadman are involved as investigators at a participating centre in the ongoing trial on the safety and efficacy of cholest‐4‐en‐3‐one, oxime for children with SMA types II and IIIa (NCT01302600). They do not receive any funding from the pharmaceutical industry.
Dr Bosboom has no conflict of interest.
Dr Wokke has no conflict of interest.
Acknowledgements
Editorial support from the Cochrane Neuromuscular Disease Group was funded for the original review by the TREAT NMD European Union Grant 036825. The Cochrane Neuromuscular Disease Group editorial base is supported by the MRC Centre for Neuromuscular Disease and the Muscular Dystrophy Campaign.
Version history
| Published | Title | Stage | Authors | Version |
| 2020 Jan 06 | Drug treatment for spinal muscular atrophy types II and III | Review | Renske I Wadman, W Ludo van der Pol, Wendy MJ Bosboom, Fay‐Lynn Asselman, Leonard H van den Berg, Susan T Iannaccone, Alexander FJE Vrancken | |
| 2012 Apr 18 | Drug treatment for spinal muscular atrophy types II and III | Review | Renske I Wadman, Wendy MJ Bosboom, W Ludo van der Pol, Leonard H van den Berg, John HJ Wokke, Susan T Iannaccone, Alexander FJE Vrancken | |
| 2011 Dec 07 | Drug treatment for spinal muscular atrophy types II and III | Review | Renske I Wadman, Wendy MJ Bosboom, Leonard H van den Berg, John HJ Wokke, Susan T Iannaccone, Alexander FJE Vrancken | |
| 2009 Jan 21 | Drug treatment for spinal muscular atrophy types II and III | Review | Wendy MJ Bosboom, Alexander FJE Vrancken, Leonard H van den Berg, John HJ Wokke, Susan T Iannaccone | |
| 2006 Oct 18 | Drug treatment for spinal muscular atrophy types II and III | Protocol | Wendy Bosboom, L H Van den Berg, Susan Iannaccone, Alexander FJE Vrancken, John HJ Wokke, LH van den Berg | |
Differences between protocol and review
The 'Risk of bias' methodology was revised in this update according to the Cochrane Handbook for Systematic Reviews of Interventions (Higgins 2008).
The search strategy was adjusted. Searches were performed from 1991 onwards because at that time genetic analysis of the SMN1 gene became widely available and could be used to establish the diagnosis of SMA.
Change in forced vital capacity (FVC), as a percentage of FVC predicted for height, was added as a secondary outcome measure. This was not stated in the original protocol but many trials used this as a measure of pulmonary function or the strength of respiratory muscles.
