@article{da7d2c2be82349a3aa443e2a55e67dd7,
title = "Spinal muscular atrophy care in the COVID-19 pandemic era",
abstract = "The coronavirus disease 2019 (COVID-19) pandemic has resulted in reorganization of healthcare settings affecting the delivery of clinical care to patients with spinal muscular atrophy (SMA). There is a concern that patients with SMA may be at increased risk of manifesting severe symptoms of COVID-19. Currently approved therapies for SMA improve survival and motor function; however, their delivery requires an increased exposure to the health system and a dedicated healthcare team. In this study, we discuss consensus recommendations pertaining to care of SMA patients during the pandemic. We highlight that SMA treatments should not be perceived as elective. Decisions regarding the delay of treatments should be made with consideration of the potential risks of COVID-19 exposure and the risk of that delay. We emphasize the importance of collaborative treatment decisions between the patient, family, and healthcare provider, considering any geographic- or institution-specific policies and precautions for COVID-19.",
keywords = "SMA, corona, epidemic, guidelines, pandemic, treatment",
author = "Aravindhan Veerapandiyan and Connolly, {Anne M.} and Finkel, {Richard S.} and Kapil Arya and Mathews, {Katherine D.} and Smith, {Edward C.} and Diana Castro and Butterfield, {Russell J.} and Parsons, {Julie A.} and Laurent Servais and Nancy Kuntz and Rao, {Vamshi K.} and Brandsema, {John F.} and Eugenio Mercuri and Emma Ciafaloni",
note = "Funding Information: A.V. has served on advisory boards for Biogen, PTC therapeutics, and AveXis and serves as an associate editor for neuromuscular disorders at Medlink Neurology. A.V. serves as principal investigator and sub‐investigator at Arkansas Children{\textquoteright}s Hospital for studies in DMD and SMA. A.M.C. serves on advisory boards for Sarepta, AveXis, Roche, and Acceleron. She serves on a DSMB (Catabasis) and serves as Sub‐Investigator at Nationwide Children{\textquoteright}s Hospital for studies in DMD (Sarepta) and SMA (AveXis). R.S.F. has received personal compensation for activities with Ionis Pharmaceuticals, Biogen, AveXis, Capricor, Catabasis, Lilly, Roche, Novartis; and the SMA Foundation, SMA Europe and Cure SMA as a consultant or advisor. R.S.F. has received research support from Ionis Pharmaceuticals, Biogen, Lilly, Cytokinetics Sarepta, NIH, MDA, and Summit. K.A. serves as site principal investigator for AveXis RESTORE registry for SMA. K.D.M. serves as site principal investigator for AveXis RESTORE registry for SMA. K.D.M. receives research funding from NIH, CDC, Friedreich{\textquoteright}s ataxia research alliance, MDA. K.D.M. serves as site principal investigator for studies involving Sarepta, Retrotope, Reata, PTC, Italfarmaco, Santhera, Catabasis, CSL Behring, and BMS. E.C.S. has received research support and consulting fees from AveXis and Biogen. D.C. has served in advisory boards for AveXis, Biogen, PTC therapeutics, Genentech, and Sarepta. D.C. serves in medical advisory boards for Cure SMA, GBS‐CIDP Foundation, and MGF. D.C. receives research funding from AveXis, Biogen, Fibrogen, PTC, Reveragen, and Sarepta. R.J.B. has received personal compensation for serving on the scientific advisory boards of Biogen and Sarepta. RJB has received research/grant support as principal investigator of studies from Acceleron, AveXis, Biogen, Capricor Catabasis, National Institutes of Health and National Institute of Neurological Disorders and Stroke, Pfizer, PTC, and Sarepta. J.A.P. has received compensation / research support from Biogen, AveXis, Genentech, Scholar Rock, Sarepta, and PTC. L.S. has received consulting fees from Roche, Biogen, AveXis, Cytokinetics, Audentes, Sarepta, Pfizer, Biphytis, PTC therapeutics, and Lupin. N.K. reports personal fees for participation in medical advisory boards for Audentes, AveXis, Biogen, Cytokinetics and Sarepta. MK has received support from Novartis and Biogen. V.K.R. has received support for consultation with Avexis, Biogen, Sarepta, and PTC therapeutics. J.F.B. reports consulting for Alexion, Audentes, AveXis, Biogen, Cytokinetics, Genentech, PTC Therapeutics, Sarepta, and WaVe and has received research funding as a site investigator from Alexion, AveXis, Biogen, CSL Behring, Cytokinetics, Fibrogen, Pfizer, PTC Therapeutics, Sarepta, Summit, and WaVe. E.M. has received funding as a member of advisory boards for SMA studies for AveXis, Biogen, Ionis Pharmaceuticals, Novartis, Scholar Rock and Roche. E.M. has served as principal investigator for Ionis Pharmaceuticals, Biogen, AveXis and Roche clinical trials. Has received funding from Famiglie SMA Italy, Italian Telethon, Biogen, and SMA Europe E.C. has received personal compensation for serving on advisory boards and/or as a consultant for AveXis, Biogen, Medscape, Pfizer, PTC Therapeutics, Sarepta, Ra pharma, Wave, the Patient‐Centered Outcomes Research Institute and Strong bridge Biopharma. E.C. has received personal compensation for serving on a speaker{\textquoteright}s bureau for Biogen. E.C. has received research and/or grant support from the Centers for Disease Control and Prevention, Cure SMA, Muscular Dystrophy Association, National Institutes of Health, Parent Project Muscular Dystrophy, PTC Therapeutics, Santhera, Sarepta Therapeutics, Orphazyme, and the US Food and Drug Administration. E.C. has received royalties from Oxford University Press and compensation from for editorial duties. Medlink Publisher Copyright: {\textcopyright} 2020 Wiley Periodicals, Inc.",
year = "2020",
month = jul,
day = "1",
doi = "10.1002/mus.26903",
language = "English (US)",
volume = "62",
pages = "46--49",
journal = "Muscle and Nerve",
issn = "0148-639X",
publisher = "John Wiley and Sons Inc.",
number = "1",
}