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January 09, 2018; 90 (2) Views & Reviews

Recommendations of the Global Multiple System Atrophy Research Roadmap Meeting

Ryan R. Walsh, Florian Krismer, Wendy R. Galpern, Gregor K. Wenning, Phillip A. Low, Glenda Halliday, Walter J. Koroshetz, Janice Holton, Niall P. Quinn, Olivier Rascol, Leslie M. Shaw, David Eidelberg, Pam Bower, Jeffrey L. Cummings, Victor Abler, Judy Biedenharn, Gal Bitan, David J. Brooks, Patrik Brundin, Hubert Fernandez, Philip Fortier, Roy Freeman, Thomas Gasser, Art Hewitt, Günter U. Höglinger, Matt J. Huentelman, Poul H. Jensen, Andreas Jeromin, Un Jung Kang, Horacio Kaufmann, Lawrence Kellerman, Vikram Khurana, Thomas Klockgether, Woojin Scott Kim, Carol Langer, Peter LeWitt, Eliezer Masliah, Wassilios Meissner, Ronald Melki, Susanne Ostrowitzki, Steven Piantadosi, Werner Poewe, David Robertson, Cyndi Roemer, Dale Schenk, Michael Schlossmacher, Jeremy D. Schmahmann, Klaus Seppi, Lily Shih, Andrew Siderowf, Glenn T. Stebbins, Nadia Stefanova, Shoji Tsuji, Sharon Sutton, Jing Zhang
First published December 13, 2017, DOI: https://doi.org/10.1212/WNL.0000000000004798
Ryan R. Walsh
From Muhammad Ali Parkinson Center (R.R.W.), Barrow Neurological Institute, Phoenix, AZ; Department of Neurology (F.K., G.K.W., W.P., K.S., N.S.), Medical University Innsbruck, Austria; National Institute of Neurological Disorders and Stroke (W.R.G., W.J.K.), NIH, Bethesda, MD; Department of Neurology (P.A.L.), Mayo Clinic, Rochester, MN; Brain and Mind Centre (G.H.), Sydney Medical School, the University of Sydney; UNSW Medicine & Neuroscience Research Australia (G.H.), Sydney; Queen Square Brain Bank (J.H.), UCL Institute of Neurology (N.P.Q.), London, UK; UPS, INSERM, and CHU de Toulouse (O.R.), University de Toulouse; Centre de Référence Maladie Rare contre l'AMS (O.R.), Centre d'Investigation Clinique CIC1436, Départments de Pharmacologie Clinique et de Neurosciences, NeuroToul/COEN Center of Excellence in Neurodegeneration, Toulouse, France; Department of Pathology & Laboratory Medicine (L.M.S.), Perelman School of Medicine, University of Pennsylvania, Philadelphia; Center for Neurosciences (D.E.), The Feinstein Institute for Medical Research, Manhasset, NY; The Multiple System Atrophy (MSA) Coalition, Inc. (P. B., J.B., P.F., L.K., C.L., C.R., S.S.), Charlotte, NC; Cleveland Clinic Lou Ruvo Center for Brain Health (J.L.C.), Las Vegas, NV; Medical Affairs (V.A.), Teva Pharmaceuticals, Frazer, PA; Department of Neurology (G.B.), David Geffen School of Medicine, Brain Research Institute, and Molecular Biology Institute, University of California at Los Angeles; Institute of Neurology (D.J.B.), University of Newcastle upon Tyne, UK; Van Andel Research Institute (P. Brundin), Center for Neurodegenerative Science, Grand Rapids, MI; Center for Neurological Restoration (H.F.), Cleveland Clinic, OH; Defeat MSA (P.F.); MSA Shoe Charity (P.F., C.R.), Saint Clair Shores, MI; Department of Neurology (R.F.), Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA; Department of Neurodegenerative Diseases (T.G.), Hertie Institute of Clinical Brain Research, University of Tübingen; German Center for Neurodegenerative Diseases (DZNE) (T.G.), Tübingen, Germany; Lundbeck LLC (A.H.), Deerfield, IL; German Center for Neurodegenerative Diseases (DZNE) (G.U.H.), Munich; Department of Neurology (G.U.H.), Technical University of Munich, Germany; Neurogenomics Division (M.J.H.), The Translational Genomics Research Institute, Phoenix, AZ; Dandrite & Department of Biomedicine (P.H.J.), Aarhus University, Denmark; Quanterix (A.J.), Lexington, MA; Department of Neurology (U.J.K.), Columbia University Medical Center; Department of Neurology (H.K.), New York University School of Medicine, New York; Whitehead Institute for Biomedical Research (V.K.), Cambridge; Ann Romney Center for Neurologic Disease (V.K.), Department of Neurology, Brigham and Women's Hospital and Harvard Medical School, Boston; Harvard Stem Cell Institute (V.K.), Cambridge, MA; Department of Neurology (T.K.), University of Bonn; German Center for Neurodegenerative Diseases (DZNE) (T.K.), Bonn, Germany; Brain and Mind Centre (W.S.K.), Sydney Medical School, University of Sydney, Australia; Department of Neurology (P.L.), Henry Ford Hospital; Department of Neurology (P.L.), Wayne State University School of Medicine, Detroit, MI; Division of Neurosciences (E.M.), National Institute on Aging/NIH, Bethesda, MD; Centre de Référence Maladie Rare AMS (W.M.), Hôpital Pellegrin, CHU de Bordeaux; Université de Bordeaux (W.M.), Institut des Maladies Neurodégénératives, UMR 5293, Bordeaux; Paris Saclay Institute of Neuroscience (R.M.), CNRS, Gif-sur-Yvette, France; Genentech Inc. (S.O.), South San Francisco; Cedars-Sinai Medical Center (S.P.), Los Angeles, CA; Department of Neurology (D.R.), Vanderbilt University Medical Center, Nashville, TN; MSA NJ (C.R.), Howell, NJ; Prothena Biosciences (D.S.), South San Francisco, CA; Division of Neurology (M.S.), Department of Medicine, The Ottawa Hospital, Canada; Department of Neurology (J.D.S.), Massachusetts General Hospital, Harvard Medical School, Boston, MA; Cure MSA Foundation (L.S.), Fremont, CA; Avid Radiopharmaceuticals (A.S.), Philadelphia, PA; Department of Neurological Sciences (G.T.S.), Rush University Medical Center, Chicago, IL; Department of Neurology (S.T.), Graduate School of Medicine, Tokyo University, Tokyo, Japan; and University of Washington (J.Z.), Seattle. W.R.G. is currently affiliated with Janssen Research and Development, Titusville, NJ.
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Florian Krismer
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Wendy R. Galpern
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Gregor K. Wenning
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Phillip A. Low
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Glenda Halliday
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Walter J. Koroshetz
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Janice Holton
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Niall P. Quinn
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Olivier Rascol
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Leslie M. Shaw
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Citation
Recommendations of the Global Multiple System Atrophy Research Roadmap Meeting
Ryan R. Walsh, Florian Krismer, Wendy R. Galpern, Gregor K. Wenning, Phillip A. Low, Glenda Halliday, Walter J. Koroshetz, Janice Holton, Niall P. Quinn, Olivier Rascol, Leslie M. Shaw, David Eidelberg, Pam Bower, Jeffrey L. Cummings, Victor Abler, Judy Biedenharn, Gal Bitan, David J. Brooks, Patrik Brundin, Hubert Fernandez, Philip Fortier, Roy Freeman, Thomas Gasser, Art Hewitt, Günter U. Höglinger, Matt J. Huentelman, Poul H. Jensen, Andreas Jeromin, Un Jung Kang, Horacio Kaufmann, Lawrence Kellerman, Vikram Khurana, Thomas Klockgether, Woojin Scott Kim, Carol Langer, Peter LeWitt, Eliezer Masliah, Wassilios Meissner, Ronald Melki, Susanne Ostrowitzki, Steven Piantadosi, Werner Poewe, David Robertson, Cyndi Roemer, Dale Schenk, Michael Schlossmacher, Jeremy D. Schmahmann, Klaus Seppi, Lily Shih, Andrew Siderowf, Glenn T. Stebbins, Nadia Stefanova, Shoji Tsuji, Sharon Sutton, Jing Zhang
Neurology Jan 2018, 90 (2) 74-82; DOI: 10.1212/WNL.0000000000004798

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Abstract

Multiple system atrophy (MSA) is a rare neurodegenerative disorder with substantial knowledge gaps despite recent gains in basic and clinical research. In order to make further advances, concerted international collaboration is vital. In 2014, an international meeting involving leaders in the field and MSA advocacy groups was convened in Las Vegas, Nevada, to identify critical research areas where consensus and progress was needed to improve understanding, diagnosis, and treatment of the disease. Eight topic areas were defined: pathogenesis, preclinical modeling, target identification, endophenotyping, clinical measures, imaging biomarkers, nonimaging biomarkers, treatments/trial designs, and patient advocacy. For each topic area, an expert served as a working group chair and each working group developed priority-ranked research recommendations with associated timelines and pathways to reach the intended goals. In this report, each groups' recommendations are provided.

Glossary

α-syn=
α-synuclein;
MSA=
multiple system atrophy;
SWOT=
strengths, weaknesses, opportunities, and threats

Footnotes

  • ↵* These authors contributed equally to this work.

  • Go to Neurology.org/N for full disclosures. Funding information and disclosures deemed relevant by the authors, if any, are provided at the end of the article.

  • Received April 24, 2017.
  • Accepted in final form September 29, 2017.
  • Copyright © 2017 American Academy of Neurology
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