Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adults. Consensus recommendations

Type of publication: Guidelines, Outcome measures, Workshop report
Disease: Mitochondrial disease
Authors:

Mancuso M, McFarland R, Klopstock T, Hirano M; consortium on Trial Readiness in Mitochondrial Myopathies.

Twenty-six researchers from 10 different countries (USA, Spain, Italy, France, Germany, The Netherlands, United Kingdom, Japan, Norway and Canada) met in Rome, Italy, from 16–18 November 2016 to update current knowledge on clinical trial readiness and outcome measures for Primary Mitochondrial Myopathies (PMM). Patients’ advocacy groups delegates also attended.

Published: 12 December 2017
Journal: Neuromuscular Disorders, volume 27, issue 12, pages 1126-1137

Link: doi.org/doi.org/10.1016/j.nmd.2017.08.006

This publication is recommended by the Mitochondrial Diseases Working Group group.

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