摘要
PM&REarly View CLINICAL LETTER Inpatient rehabilitation admission for a patient with spinal muscular atrophy status post gene therapy Jaclyn C. Omura MD, Corresponding Author Jaclyn C. Omura MD [email protected] orcid.org/0000-0001-5416-0956 Rehabilitation Medicine, University of Washington School of Medicine, Seattle Children's Hospital, Seattle, Washington, USA Correspondence Jaclyn C. Omura, Rehabilitation Medicine, University of Washington School of Medicine, Seattle Children's Hospital, 4800 Sand Point Way, Seattle, WA 98105, USA. Email: [email protected]Search for more papers by this authorCasey L. Freeman MD, Casey L. Freeman MD Physical Medicine and Rehabilitation, University of Colorado Health, Greeley, Colorado, USASearch for more papers by this authorSeth J. Perlman MD, Seth J. Perlman MD Neurology, University of Washington School of Medicine, Seattle Children's Hospital, Seattle, Washington, USASearch for more papers by this authorMolly M. Fuentes MD, MS, Molly M. Fuentes MD, MS Rehabilitation Medicine, University of Washington School of Medicine, Seattle Children's Hospital, Seattle, Washington, USASearch for more papers by this author Jaclyn C. Omura MD, Corresponding Author Jaclyn C. Omura MD [email protected] orcid.org/0000-0001-5416-0956 Rehabilitation Medicine, University of Washington School of Medicine, Seattle Children's Hospital, Seattle, Washington, USA Correspondence Jaclyn C. Omura, Rehabilitation Medicine, University of Washington School of Medicine, Seattle Children's Hospital, 4800 Sand Point Way, Seattle, WA 98105, USA. Email: [email protected]Search for more papers by this authorCasey L. Freeman MD, Casey L. Freeman MD Physical Medicine and Rehabilitation, University of Colorado Health, Greeley, Colorado, USASearch for more papers by this authorSeth J. Perlman MD, Seth J. Perlman MD Neurology, University of Washington School of Medicine, Seattle Children's Hospital, Seattle, Washington, USASearch for more papers by this authorMolly M. Fuentes MD, MS, Molly M. Fuentes MD, MS Rehabilitation Medicine, University of Washington School of Medicine, Seattle Children's Hospital, Seattle, Washington, USASearch for more papers by this author First published: 19 January 2023 https://doi.org/10.1002/pmrj.12948 We obtained written consent from this patient's family prior to writing this manuscript. Read the full textAboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onEmailFacebookTwitterLinkedInRedditWechat No abstract is available for this article. REFERENCES 1Finkel RS, McDermott MP, Kaufmann P, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014; 83(9): 810-817. 2Calucho M, Bernal S, Alías L, et al. Correlation between SMA type and SMN2 copy number revisited: an analysis of 625 unrelated Spanish patients and a compilation of 2834 reported cases. Neuromuscular Disord. 2018; 28(3): 208-215. 3https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease. 4Mendell JR, Al-Zaidy S, Shell R, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med. 2017; 377(18): 1713-1722. 5Pera MC, Coratti G, Forcina N, et al. Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy. BMC Neurol. 2017; 17(1): 1-10. 6Waldrop MA, Karingada C, Storey MA, et al. Gene therapy for spinal muscular atrophy: safety and early outcomes. Pediatrics. 2020; 146(3). 7Lohse KR, Lang CE, Boyd LA. Is more better? Using metadata to explore dose–response relationships in stroke rehabilitation. Stroke. 2014; 45(7): 2053-2058. Early ViewOnline Version of Record before inclusion in an issue ReferencesRelatedInformation