@article{fdc5936700084f0b91a7d41a7d7ab44f,
title = "Primary lateral sclerosis (PLS) functional rating scale: PLS-specific clinimetric scale",
abstract = "Introduction: Our research aim was to develop a novel clinimetric scale sensitive enough to detect disease progression in primary lateral sclerosis (PLS). Methods: A prototype of the PLS Functional Rating Scale (PLSFRS) was generated. Seventy-seven participants with PLS were enrolled and evaluated at 21 sites that comprised the PLSFRS study group. Participants were assessed using the PLSFRS, Neuro-Quality of Life (QoL), Schwab-England Activities of Daily Living (ADL), and the Clinical Global Impression of Change scales. Participants completed telephone assessments at 12, 24, and 48 weeks after enrollment. Results: The PLSFRS demonstrated internal consistency as well as intrarater, interrater, telephone test-retest reliability, and construct validity. Significant changes in disease progression were detected at 6 and 12 months; changes measured by the PLSFRS vs the ALSFRS-R were significantly higher. Discussion: The PLSFRS is a valid tool to assess the natural history of PLS in a shorter study period.",
keywords = "MND, PLS, PLSFRS, PUMND, clinical trials, clinimetric scale",
author = "{the PLSFRS study group} and Hiroshi Mitsumoto and Codruta Chiuzan and Madison Gilmore and Yuan Zhang and Zachary Simmons and Sabrina Paganoni and Kisanuki, {Yasushi Y.} and Lorne Zinman and Omar Jawdat and Eric Sorenson and Floeter, {Mary Kay} and Pioro, {Erik P.} and {Fernandes Filho}, {J. Americo M.} and Daragh Heitzman and Fournier, {Christina Nicole} and Bjorn Oskarsson and Terry Heiman-Patterson and Nicholas Maragakis and Nanette Joyce and Ghazala Hayat and Sharon Nations and Stephen Scelsa and David Walk and Lauren Elman and Jonathan Hupf and Brittany McHale",
note = "Funding Information: H.M. has received funding from the NIH, the CDC, the MDA, the ALS Association, the MNDA, the SPF, Tsumura, Cytokinetics, Mitsubishi‐Tanabe; funding for clinical trials from Cytokinetics; and has been on the advisory boards for Mitsubishi‐Tanabe, Biohaven, and Daihippon‐Sumitomo. Z.S. has served as a consultant for Biohaven; received research support from Biohaven, Mallinckrodt, and Sanofi; and receives a stipend from Wiley as Editor and Chief for Muscle & Nerve . S.P. has received research grants from Target ALS, the Salah Foundation, the Spastic Paraplegia Foundation, the ALS Association, ALS Finding a Cure, the American Academy of Neurology, Amylyx Therapeutics, and Revalesio Corporation; advisory board fees from Roche; and honoraria from Oakstone Publishing and McGraw‐Hill Education. M.K.F. has received support from the intramural program of NINDS, NIH. E.P.P. has received clinical trial and research funding from the NIH/CDC and the ALS Association; consulting fees from Avanir Pharmaceuticals, Biohaven Pharmaceuticals, Cytokinetics, ITF Pharma, MT Pharma America, and Otsuka America. J.A.M.F. has received support for clinical trials from Mallinckrodt Pharmaceuticals. D.H. has received speaker/consultant fees from Biohaven Pharmaceuticals, and research grant support from Cytokinetics, Mallinckrodt Pharmaceuticals, Orion Pharma, Amylyx Therapeutics, Revance Therapeutics, the MDA, the ALS Association, and the Neurologix Foundation. C.N.F. has received research funding from the Department of Veterans Affairs Office of Research and Development (IK2CX001595–02), Mallinckrodt ARD, and Amylyx Pharmaceuticals. B.O. has received grants from Target ALS, NINDS, Biogen, Genentech, Cytokinetics, Orion, Eisai, and consulting/advisory board fees from Mitsubishi Tanabe, Medicinova, and Biohaven. T.H.‐P. has received consultant and advisory board fees from Mitsubishi Tanabe Pharma, Cytokinetics, ITF, and Biohaven. N.M. has received consultant/advisory board fees from Biogen, Clene Nanomedicine, Apellis, Brainstorm, Orion Pharma; DSMC from Orphazyme; and grant support from the ALS Association, the Department of Defense ALSRP, the NINDS, and the Maryland Stem Cell Research Fund. N.J. has received grants from the NIH, NIDDR; clinical trials funding from Novartis, Acceleron, Mitsubishi‐Tanabe; and speaker/consultant fees from Biogen. D.W. has been a consultant for MT Pharma and Sarepta Therapeutics. The remaining authors declare no conflicts of interest. Funding Information: information Spastic Paraplegia Foundation (research grant); David Marren and familyThe authors are grateful to the participants and their families for being part of this clinical study. We thank the study funders for their contributions to the development of the PLSFRS scale. Georgia Christodoulou, MA, University of Southern California, assisted with the preparation of the manuscript and editing. Publisher Copyright: {\textcopyright} 2019 Wiley Periodicals, Inc.",
year = "2020",
month = feb,
day = "1",
doi = "10.1002/mus.26765",
language = "English (US)",
volume = "61",
pages = "163--172",
journal = "Muscle and Nerve",
issn = "0148-639X",
publisher = "John Wiley and Sons Inc.",
number = "2",
}