TY - JOUR
T1 - Recommendations of the Global Multiple System Atrophy Research Roadmap Meeting
AU - Walsh, Ryan R.
AU - Krismer, Florian
AU - Galpern, Wendy R.
AU - Wenning, Gregor K.
AU - Low, Phillip A.
AU - Halliday, Glenda
AU - Koroshetz, Walter J.
AU - Holton, Janice
AU - Quinn, Niall P.
AU - Rascol, Olivier
AU - Shaw, Leslie M.
AU - Eidelberg, David
AU - Bower, Pam
AU - Cummings, Jeffrey L.
AU - Abler, Victor
AU - Biedenharn, Judy
AU - Bitan, Gal
AU - Brooks, David J.
AU - Brundin, Patrik
AU - Fernandez, Hubert
AU - Fortier, Philip
AU - Freeman, Roy
AU - Gasser, Thomas
AU - Hewitt, Art
AU - Höglinger, Günter U.
AU - Huentelman, Matt J.
AU - Jensen, Poul H.
AU - Jeromin, Andreas
AU - Kang, Un Jung
AU - Kaufmann, Horacio
AU - Kellerman, Lawrence
AU - Khurana, Vikram
AU - Klockgether, Thomas
AU - Kim, Woojin Scott
AU - Langer, Carol
AU - Lewitt, Peter
AU - Masliah, Eliezer
AU - Meissner, Wassilios
AU - Melki, Ronald
AU - Ostrowitzki, Susanne
AU - Piantadosi, Steven
AU - Poewe, Werner
AU - Robertson, David
AU - Roemer, Cyndi
AU - Schenk, Dale
AU - Schlossmacher, Michael
AU - Schmahmann, Jeremy D.
AU - Seppi, Klaus
AU - Shih, Lily
AU - Siderowf, Andrew
AU - Stebbins, Glenn T.
AU - Stefanova, Nadia
AU - Tsuji, Shoji
AU - Sutton, Sharon
AU - Zhang, Jing
N1 - Funding Information:
disclosures relevant to the manuscript. T. Klockgether receives/ has received research support from the Deutsche For-schungsgemeinschaft (DFG), the Bundesministerium für Bil-dung und Forschung (BMBF), the Bundesministerium für Gesundheit (BMG), the Robert Bosch Foundation, and the European Union (EU). He serves on the editorial board of The Cerebellum and the Journal of Neurology. He has received consulting fees from Biohaven, ICON Clinical Research, and Atheneum. W.S. Kim, C. Langer, P. LeWitt, E. Masliah, W. Meissner, and R. Melki report no disclosures relevant to the manuscript. S. Ostrowitzki is an employee of Genentech Inc., a Member of the Roche Group. S. Piantadosi reports no disclosures relevant to the manuscript. W. Poewe reports personal fees from AbbVie, Allergan, AstraZeneca, Boehringer-Ingelheim, Boston Scientific, Britannia, GlaxoSmithKline, Ipsen, Lundbeck, Medtronic, MSD, Merck-Serono, Merz Pharmaceuticals, Novartis, Orion Pharma, Teva, UCB, and Zambon (consultancy and lecture fees in relation to clinical drug development programs for PD). Royalties: Thieme, Wiley Blackwell, Oxford University Press, and Cambridge University Press. D. Robertson, C. Roemer, D. Schenk, M. Schlossmacher, J.D. Schmahmann, K. Seppi, L. Shih, and A. Siderowf report no disclosures relevant to the manuscript. G.T. Stebbins: consulting and advisory board membership with honoraria: Acadia Pharmaceuticals, Adamas Pharmaceuticals, Inc., Ceregene, Inc., CHDI Management, Inc., Ingenix Pharmaceutical Services (i3 Research), Neurocrine Biosciences, Inc., Pfizer, Inc., Ultragenyx Pharmaceutical. Grants and research: NIH, Michael J. Fox Foundation for Parkinson’s Research, Dystonia Coalition, CHDI, International Parkinson and Movement Disorder Society, CBD Solutions. Honoraria: International Parkinson and Movement Disorder Society, American Academy of Neurology, Michael J. Fox Foundation for Parkinson’s Research, Food and Drug Administration. Intellectual property rights: none. Ownership interests: none. Royalties: none. Expert testimony: none. Salary: Rush University Medical Center. N. Stefanova received research grants from the Austrian Science Fund (FWF), the MSA Coalition, AstraZeneca, and Bio-Arctic Neuroscience AB. S. Tsuji, S. Sutton, and J. Zhang report no disclosures relevant to the manuscript. Go to Neurology.org/N for full disclosures.
Publisher Copyright:
© 2017 American Academy of Neurology.
PY - 2018/1/9
Y1 - 2018/1/9
N2 - 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.
AB - 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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U2 - 10.1212/WNL.0000000000004798
DO - 10.1212/WNL.0000000000004798
M3 - Article
C2 - 29237794
AN - SCOPUS:85044093997
SN - 0028-3878
VL - 90
SP - 74
EP - 82
JO - Neurology
JF - Neurology
IS - 2
ER -