Journal
NATURE REVIEWS NEUROLOGY
Volume 9, Issue 8, Pages 474-481Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nrneurol.2013.129
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Funding
- Canadian Institutes of Health Research
- Sigrid Juselius Foundation
- Jane and Atos Erkko Foundation
- DoH/HEFCE
- Eurostars Programme [ESTAR 11205]
- ZonMW PM Rare
- Dutch Science Organisation (NWO) CSBR Program [853.00.130]
- Wellcome Trust Centre for Mitochondrial Research [096919Z/11/Z]
- Medical Research Council (UK) Centre for Translational Research in Neuromuscular Diseases
- EU FP7 TIRCON
- NIHR Newcastle Biomedical Research Centre based at Newcastle upon Tyne Hospital NHS Foundation Trust
- NIHR Newcastle Biomedical Research Centre based at Newcastle upon Tyne Hospital Newcastle University
- Marriott Foundation
- Telethon Italy [GPP10005]
- German Federal Ministry of Education and Research (BMBF) [01GM1113A]
- BMBF (German Centre for Vertigo and Balance Disorders) [01E00901]
- European Commission Seventh Framework Programme (FP7, HEALTH-F2-2011) [277984]
- Medical Research Council [MC_UP_1002/1, G0701386, G1002570, G0700718, G1000848, MR/K000608/1] Funding Source: researchfish
- National Institute for Health Research [NF-SI-0509-10011] Funding Source: researchfish
- MRC [G0701386, MC_UP_1002/1, G1002570, G0800674, G1000848, MR/K000608/1, G0700718] Funding Source: UKRI
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Mitochondrial dysfunction is a common cause of inherited multisystem disease that often involves the nervous system. Despite major advances in our understanding of the pathophysiology of mitochondrial diseases, clinical management of these conditions remains largely supportive. Using a systematic approach, we identified 1,039 publications on treatments for mitochondrial diseases, only 35 of which included observations on more than five patients. Reports of a positive outcome on the basis of a biomarker of unproven clinical significance were more common in nonrandomized and nonblinded studies, suggesting a publication bias toward positive but poorly executed studies. Although trial design is improving, there is a critical need to develop new biomarkers of mitochondrial disease. In this Perspectives article, we make recommendations for the design of future treatment trials in mitochondrial diseases. Patients and physicians should no longer rely on potentially biased data, with the associated costs and risks.
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