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

Type of publication: Clinical trial readiness, Guidelines, Outcome measures
Disease: Mitochondrial disease

Authors:

Michelangelo Mancuso, Robert McFarland, Thomas Klopstock, Michio Hirano, on behalf of the consortium on Trial Readiness in Mitochondrial Myopathies, Rafael Artuch, Enrico Bertini, Laurence Bindoff, Valerio Carelli, Grainne Gorman, Michio Hirano, Rita Horvath, Petra Kaufmann, Thomas Klopstock, Yasutoshi Koga, Saskia Koene, Costanza Lamperti, Robert McFarland, Michelangelo Mancuso, Julio Montoya, Francisco Javier Pérez-Mínguez Caneda, Vincent Procaccio, Holger Prokisch, Shamima Rahman, Piero Santantonio, Markus Schülke, Serenella Servidei, Dikoma C.Shungu, Gabriele Siciliano, Jan Smeitink, Tanja Taivassalo, John L.P. Thompson Doug Turnbull, EljaVan der Veer, Philip E.Yeske, Massimo Zeviani


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

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

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