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Outcome measures for children with mitochondrial disease: consensus recommendations for future studies from a Delphi‐based international workshop

19 Jul 2018

Abstract excerpt

Although there are no effective disease-modifying therapies for mitochondrial diseases, an increasing number of trials are being conducted in this rare disease group. The use of sensitive and valid endpoints is essential to test the effectiveness of potential treatments. There is no consensus on which outcome measures to use in children with mitochondrial disease. The aims of this two-day Delphi-based workshop...

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PMID: 30027425
DOI: 10.1007/s10545-018-0229-5

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