International Workshop:: Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adults. Consensus recommendations. 16–18 November 2016, Rome, Italy

•Outcome measures and clinical trial readiness are needed in mitochondrial diseases.•Primary mitochondrial myopathy has been defined clinically and genetically.•This consensus supplies guidance to clinical researchers and industry.

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Veröffentlicht in:	Neuromuscular disorders : NMD 2017-12, Vol.27 (12), p.1126-1137
Hauptverfasser:	Mancuso, Michelangelo, McFarland, Robert, Klopstock, Thomas, Hirano, Michio, Artuch, Rafael, Bertini, Enrico, Bindoff, Laurence, Carelli, Valerio, Gorman, Grainne, Horvath, Rita, Kaufmann, Petra, Koga, Yasutoshi, Koene, Saskia, Lamperti, Costanza, Montoya, Julio, Javier Pérez-Mínguez Caneda, Francisco, Procaccio, Vincent, Prokisch, Holger, Rahman, Shamima, Santantonio, Piero, Schülke, Markus, Servidei, Serenella, Shungu, Dikoma C., Siciliano, Gabriele, Smeitink, Jan, Taivassalo, Tanja, Thompson, John L.P., Turnbull, Doug, Van der Veer, Elja, Yeske, Philip E., Zeviani, Massimo
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Child Clinical Trials as Topic - methods Congresses as Topic Consensus Humans Italy Mitochondrial Myopathies - drug therapy Outcome Assessment, Health Care - methods Outcome Assessment, Health Care - standards
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Beschreibung
Zusammenfassung:	•Outcome measures and clinical trial readiness are needed in mitochondrial diseases.•Primary mitochondrial myopathy has been defined clinically and genetically.•This consensus supplies guidance to clinical researchers and industry.
ISSN:	0960-8966 1873-2364 1873-2364
DOI:	10.1016/j.nmd.2017.08.006