TY - JOUR
T1 - Planning for Prevention of Parkinson Disease
T2 - Now Is the Time
AU - Crotty, Grace F.
AU - Keavney, Jessi L.
AU - Alcalay, Roy N.
AU - Marek, Kenneth
AU - Marshall, Gad A.
AU - Rosas, H. Diana
AU - Schwarzschild, Michael A.
N1 - Publisher Copyright:
© American Academy of Neurology.
PY - 2022/8/16
Y1 - 2022/8/16
N2 - Parkinson disease (PD) is a chronic progressive neurodegenerative disease with increasing worldwide prevalence. Despite many trials of neuroprotective therapies in manifest PD, no disease-modifying therapy has been established. Over the past several decades, a series of breakthroughs have identified discrete populations at substantially increased risk of developing PD. Based on this knowledge, now is the time to design and implement PD prevention trials. This endeavor builds on experience gained from early prevention trials in Alzheimer disease and Huntington disease. This article first reviews prevention trial precedents in these other neurodegenerative diseases before focusing on the critical design elements for PD prevention trials, including whom to enroll for these trials, what therapeutics to test, and how to measure outcomes in prevention trials. Our perspective reflects progress and remaining challenges that motivated a 2021 conference, "Planning for Prevention of Parkinson: A Trial Design Symposium and Workshop."
AB - Parkinson disease (PD) is a chronic progressive neurodegenerative disease with increasing worldwide prevalence. Despite many trials of neuroprotective therapies in manifest PD, no disease-modifying therapy has been established. Over the past several decades, a series of breakthroughs have identified discrete populations at substantially increased risk of developing PD. Based on this knowledge, now is the time to design and implement PD prevention trials. This endeavor builds on experience gained from early prevention trials in Alzheimer disease and Huntington disease. This article first reviews prevention trial precedents in these other neurodegenerative diseases before focusing on the critical design elements for PD prevention trials, including whom to enroll for these trials, what therapeutics to test, and how to measure outcomes in prevention trials. Our perspective reflects progress and remaining challenges that motivated a 2021 conference, "Planning for Prevention of Parkinson: A Trial Design Symposium and Workshop."
UR - http://www.scopus.com/inward/record.url?scp=85136525758&partnerID=8YFLogxK
U2 - 10.1212/WNL.0000000000200789
DO - 10.1212/WNL.0000000000200789
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C2 - 36219787
AN - SCOPUS:85136525758
SN - 0028-3878
VL - 99
SP - S1-S9
JO - Neurology
JF - Neurology
IS - 7
ER -