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

Michelangelo Mancuso¹, Robert McFarland², Thomas Klopstock³, Michio Hirano⁴; consortium on Trial Readiness in Mitochondrial Myopathies

Collaborators, Affiliations

Collaborators

consortium on Trial Readiness in Mitochondrial Myopathies:
Rafael Artuch⁵, Enrico Bertini⁶, Laurence Bindoff⁷, Valerio Carelli⁸, Grainne Gorman⁹, Michio Hirano¹⁰, Rita Horvath⁹, Petra Kaufmann¹¹, Thomas Klopstock¹², Yasutoshi Koga¹³, Saskia Koene¹⁴, Costanza Lamperti¹⁵, Robert McFarland⁹, Michelangelo Mancuso¹⁶, Julio Montoya¹⁷, Francisco Javier Pérez-Mínguez Caneda¹⁸, Vincent Procaccio¹⁹, Holger Prokisch²⁰, Shamima Rahman²¹, Piero Santantonio²², Markus Schülke²³, Serenella Servidei²⁴, Dikoma C Shungu²⁵, Gabriele Siciliano¹⁶, Jan Smeitink¹⁴, Tanja Taivassalo²⁶, John L P Thompson¹⁰, Doug Turnbull⁹, Elja Van der Veer²⁷, Philip E Yeske²⁸, Massimo Zeviani²⁹

Affiliations

¹ Department of Experimental and Clinical Medicine, Neurological Institute, University of Pisa, Italy. Electronic address: mancusomichelangelo@gmail.com.
² Wellcome Trust Centre for Mitochondrial Research, Institute of Genetic Medicine, Department of Physiology and Functional Genomics NE1 3BZ, Newcastle University, Newcastle upon Tyne, UK.
³ Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik, LMU München, Ziemssenstr. 1a, 80336 München, Federal Republic of Germany.
⁴ Department of Neurology, H. Houston Merritt Neuromuscular Research Center, Columbia University Medical Center, New York, NY, USA.
⁵ Institut de Recerca Sant Joan de Déu and CIBERER, Barcelona, Spain.
⁶ Bambino Gesù Hospital IRCCS, Rome, Italy.
⁷ University of Bergen & Haukeland University Hospital, Norway.
⁸ IRCCS Bellaria Hospital and University of Bologna, Italy.
⁹ Wellcome Trust Centre for Mitochondrial Research, Newcastle upon Tyne, UK.
¹⁰ Columbia University Medical Center, New York, USA.
¹¹ National Center for Advancing Translational Sciences, Bethesda, MD, USA.
¹² Friedrich Baur Institute, Munich, Federal Republic of Germany.
¹³ Kurume University School of Medicine, Kurume, Fukuoka 830-0011, Japan.
¹⁴ Radboud Center for Mitochondrial Medicine, Nijmegen, The Netherlands.
¹⁵ The Foundation "Carlo Besta" Institute of Neurology-IRCCS, Milan, Italy.
¹⁶ University of Pisa, Italy.
¹⁷ Universidad de Zaragoza, CIBERER, Instituto de Investigación Sanitaria de Aragón, Spain.
¹⁸ Asociación de Enfermos con Patologías Mitocondriales, Spain.
¹⁹ University of Angers, France.
²⁰ Institute of Human Genetics, Technische Universität München, Federal Republic of Germany.
²¹ UCL Great Ormond Street Institute of Child Health, London, UK.
²² MITOCON Association, Italy.
²³ Charité Campus Virchow Klinikum, Berlin, Federal Republic of Germany.
²⁴ Institute of Neurology, Catholic University, Rome, Italy.
²⁵ Weill Cornell Medicine, New York, USA.
²⁶ University of Florida, Gainesville, USA.
²⁷ President, International Mito Patients Association, The Netherlands.
²⁸ United Mitochondrial Disease Foundation (UMDF), Pittsburgh, USA.
²⁹ MRC-Mitochondrial Biology Unit, Cambridge, UK.

PMID: 29074296
PMCID: PMC6094160
DOI: 10.1016/j.nmd.2017.08.006

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

Michelangelo Mancuso et al. Neuromuscul Disord. 2017 Dec.

. 2017 Dec;27(12):1126-1137.

doi: 10.1016/j.nmd.2017.08.006. Epub 2017 Sep 8.

Authors

Michelangelo Mancuso¹, Robert McFarland², Thomas Klopstock³, Michio Hirano⁴; consortium on Trial Readiness in Mitochondrial Myopathies

Collaborators

consortium on Trial Readiness in Mitochondrial Myopathies:
Rafael Artuch⁵, Enrico Bertini⁶, Laurence Bindoff⁷, Valerio Carelli⁸, Grainne Gorman⁹, Michio Hirano¹⁰, Rita Horvath⁹, Petra Kaufmann¹¹, Thomas Klopstock¹², Yasutoshi Koga¹³, Saskia Koene¹⁴, Costanza Lamperti¹⁵, Robert McFarland⁹, Michelangelo Mancuso¹⁶, Julio Montoya¹⁷, Francisco Javier Pérez-Mínguez Caneda¹⁸, Vincent Procaccio¹⁹, Holger Prokisch²⁰, Shamima Rahman²¹, Piero Santantonio²², Markus Schülke²³, Serenella Servidei²⁴, Dikoma C Shungu²⁵, Gabriele Siciliano¹⁶, Jan Smeitink¹⁴, Tanja Taivassalo²⁶, John L P Thompson¹⁰, Doug Turnbull⁹, Elja Van der Veer²⁷, Philip E Yeske²⁸, Massimo Zeviani²⁹

Affiliations

¹ Department of Experimental and Clinical Medicine, Neurological Institute, University of Pisa, Italy. Electronic address: mancusomichelangelo@gmail.com.
² Wellcome Trust Centre for Mitochondrial Research, Institute of Genetic Medicine, Department of Physiology and Functional Genomics NE1 3BZ, Newcastle University, Newcastle upon Tyne, UK.
³ Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik, LMU München, Ziemssenstr. 1a, 80336 München, Federal Republic of Germany.
⁴ Department of Neurology, H. Houston Merritt Neuromuscular Research Center, Columbia University Medical Center, New York, NY, USA.
⁵ Institut de Recerca Sant Joan de Déu and CIBERER, Barcelona, Spain.
⁶ Bambino Gesù Hospital IRCCS, Rome, Italy.
⁷ University of Bergen & Haukeland University Hospital, Norway.
⁸ IRCCS Bellaria Hospital and University of Bologna, Italy.
⁹ Wellcome Trust Centre for Mitochondrial Research, Newcastle upon Tyne, UK.
¹⁰ Columbia University Medical Center, New York, USA.
¹¹ National Center for Advancing Translational Sciences, Bethesda, MD, USA.
¹² Friedrich Baur Institute, Munich, Federal Republic of Germany.
¹³ Kurume University School of Medicine, Kurume, Fukuoka 830-0011, Japan.
¹⁴ Radboud Center for Mitochondrial Medicine, Nijmegen, The Netherlands.
¹⁵ The Foundation "Carlo Besta" Institute of Neurology-IRCCS, Milan, Italy.
¹⁶ University of Pisa, Italy.
¹⁷ Universidad de Zaragoza, CIBERER, Instituto de Investigación Sanitaria de Aragón, Spain.
¹⁸ Asociación de Enfermos con Patologías Mitocondriales, Spain.
¹⁹ University of Angers, France.
²⁰ Institute of Human Genetics, Technische Universität München, Federal Republic of Germany.
²¹ UCL Great Ormond Street Institute of Child Health, London, UK.
²² MITOCON Association, Italy.
²³ Charité Campus Virchow Klinikum, Berlin, Federal Republic of Germany.
²⁴ Institute of Neurology, Catholic University, Rome, Italy.
²⁵ Weill Cornell Medicine, New York, USA.
²⁶ University of Florida, Gainesville, USA.
²⁷ President, International Mito Patients Association, The Netherlands.
²⁸ United Mitochondrial Disease Foundation (UMDF), Pittsburgh, USA.
²⁹ MRC-Mitochondrial Biology Unit, Cambridge, UK.

PMID: 29074296
PMCID: PMC6094160
DOI: 10.1016/j.nmd.2017.08.006

No abstract available

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References

1. Gorman GS, Chinnery PF, DiMauro S, Hirano M, Koga Y, McFarland R, et al. Mitochondrial diseases. Nat Rev Dis Primers. 2016;2:16080. - PubMed
1. Pitceathly RD, McFarland R. Mitochondrial myopathies in adults and children: management and therapy development. Curr Opin Neurol. 2014;27(5):576–82. - PubMed
1. Pfeffer G, Majamaa K, Turnbull DM, Thorburn D, Chinnery PF. Treatment for mitochondrial disorders. Cochrane Database Syst Rev. 2012;(4):CD004426. - PMC - PubMed
1. Koopman WJ, Beyrath J, Fung CW, Koene S, Rodenburg RJ, Willems PH, et al. Mitochondrial disorders in children: toward development of small-molecule treatment strategies. EMBO Mol Med. 2016;8(4):311–27. - PMC - PubMed
1. Viscomi C. Toward a therapy for mitochondrial disease. Biochem Soc Trans. 2016;44(5):1483–90. - PMC - PubMed

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

Collaborators

Affiliations

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

Authors

Collaborators

Affiliations

References

Publication types

MeSH terms

Grants and funding

LinkOut - more resources

Full Text Sources

Other Literature Sources

Medical