Recommendations of the Global Multiple System Atrophy Research Roadmap Meeting.

Neurology

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.

Published: January 2018

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.

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5772155PMC
http://dx.doi.org/10.1212/WNL.0000000000004798DOI Listing

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