Paving the Way Toward Meaningful Trials in Ataxias: An Ataxia Global Initiative Perspective

Thomas Klockgether^{1

2}, Tetsuo Ashizawa³, Bernard Brais⁴, Rosalind Chuang⁵, Alexandra Durr⁶, Brent Fogel⁷, Julie Greenfield⁸, Sue Hagen⁹, Laura Bannach Jardim^{10

11}, Hong Jiang¹², Osamu Onodera¹³, José Luiz Pedroso¹⁴, Bin-Weng Soong^{15

16}, David Szmulewicz¹⁷, Holm Graessner^{18

19}, Matthis Synofzik^{20

21}; Ataxia Global Initiative (AGI)

Affiliations

¹ German Center for Neurodegenerative Diseases (DZNE), Bonn, Germany.
² Department of Neurology, University Hospital Bonn, Bonn, Germany.
³ Houston Methodist Research Institute and Weil Cornell Medical College at Houston Methodist, Houston, Texas, USA.
⁴ McGill University, Montreal, Quebec, Canada.
⁵ Biogen, Cambridge, Massachusetts, USA.
⁶ Sorbonne Université, Paris Brain Institute, Paris Brain Institute - ICM, INSERM, CNRS, APHP, University Hospital de la Pitié-Salpêtrière Paris, Paris, France.
⁷ Departments of Neurology and Human Genetics, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, USA.
⁸ Ataxia UK, London, United Kingdom.
⁹ National Ataxia Foundation, Minneapolis, Minnesota, USA.
¹⁰ Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil.
¹¹ Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil.
¹² Xiangya Hospital, Central South University, Changsha, China.
¹³ Brain Research Institute, Niigata University, Niigata, Japan.
¹⁴ Ataxia Unit, Department of Neurology, Universidade Federal de São Paulo, São Paulo, Brazil.
¹⁵ National Yang-Ming Chiao Tung University, Taipei, Taiwan.
¹⁶ Taipei Neurologic Institute, Taipei Medical University, Taipei, Taiwan.
¹⁷ University of Melbourne, Parkville, Victoria, Australia.
¹⁸ Institute of Medical Genetics and Applied Genomics, University of Tübingen, Tübingen, Germany.
¹⁹ Center for Rare Diseases, University Hospital Tübingen, Tübingen, Germany.
²⁰ Division Translational Genomics of Neurodegenerative Diseases, Center for Neurology and Hertie-Institute for Clinical Brain Research, University of Tübingen, Tübingen, Germany.
²¹ German Center for Neurodegenerative Diseases (DZNE), Tübingen, Germany.

PMID: 35475582
DOI: 10.1002/mds.29032

Paving the Way Toward Meaningful Trials in Ataxias: An Ataxia Global Initiative Perspective

Thomas Klockgether et al. Mov Disord. 2022 Jun.

. 2022 Jun;37(6):1125-1130.

doi: 10.1002/mds.29032. Epub 2022 Apr 27.

Authors

Affiliations

¹ German Center for Neurodegenerative Diseases (DZNE), Bonn, Germany.
² Department of Neurology, University Hospital Bonn, Bonn, Germany.
³ Houston Methodist Research Institute and Weil Cornell Medical College at Houston Methodist, Houston, Texas, USA.
⁴ McGill University, Montreal, Quebec, Canada.
⁵ Biogen, Cambridge, Massachusetts, USA.
⁶ Sorbonne Université, Paris Brain Institute, Paris Brain Institute - ICM, INSERM, CNRS, APHP, University Hospital de la Pitié-Salpêtrière Paris, Paris, France.
⁷ Departments of Neurology and Human Genetics, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, USA.
⁸ Ataxia UK, London, United Kingdom.
⁹ National Ataxia Foundation, Minneapolis, Minnesota, USA.
¹⁰ Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil.
¹¹ Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil.
¹² Xiangya Hospital, Central South University, Changsha, China.
¹³ Brain Research Institute, Niigata University, Niigata, Japan.
¹⁴ Ataxia Unit, Department of Neurology, Universidade Federal de São Paulo, São Paulo, Brazil.
¹⁵ National Yang-Ming Chiao Tung University, Taipei, Taiwan.
¹⁶ Taipei Neurologic Institute, Taipei Medical University, Taipei, Taiwan.
¹⁷ University of Melbourne, Parkville, Victoria, Australia.
¹⁸ Institute of Medical Genetics and Applied Genomics, University of Tübingen, Tübingen, Germany.
¹⁹ Center for Rare Diseases, University Hospital Tübingen, Tübingen, Germany.
²⁰ Division Translational Genomics of Neurodegenerative Diseases, Center for Neurology and Hertie-Institute for Clinical Brain Research, University of Tübingen, Tübingen, Germany.
²¹ German Center for Neurodegenerative Diseases (DZNE), Tübingen, Germany.

PMID: 35475582
DOI: 10.1002/mds.29032

No abstract available

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References

1. Klockgether T, Mariotti C, Paulson HL. Spinocerebellar ataxia. Nat Rev Dis Primers 2019;5:24
1. Synofzik M, Puccio H, Mochel F, Schöls L. Autosomal recessive cerebellar ataxias: paving the way toward targeted molecular therapies. Neuron 2019;101:560-583. https://doi.org/10.1016/j.neuron.2019.01.049
1. Beaudin M, Matilla-Dueñas A, Soong B-W, et al. The classification of autosomal recessive cerebellar ataxias: a consensus statement from the Society for Research on the cerebellum and ataxias task force. Cerebellum 2019;18:1098-1125. https://doi.org/10.1007/s12311-019-01052-2
1. Vázquez-Mojena Y, León-Arcia K, González-Zaldivar Y, Rodríguez-Labrada R, Velázquez-Pérez L. Gene therapy for Polyglutamine spinocerebellar ataxias: advances, challenges, and perspectives. Mov Disord 2021;36:2731-2744. https://doi.org/10.1002/mds.28819
1. Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, et al. Targeting huntingtin expression in patients with Huntington's disease. N Engl J Med 2019;380:2307-2316.

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Paving the Way Toward Meaningful Trials in Ataxias: An Ataxia Global Initiative Perspective

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Paving the Way Toward Meaningful Trials in Ataxias: An Ataxia Global Initiative Perspective

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