{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,8]],"date-time":"2026-07-08T21:48:42Z","timestamp":1783547322308,"version":"3.55.0"},"reference-count":71,"publisher":"IMR Press","issue":"6","license":[{"start":{"date-parts":[[2022,6,6]],"date-time":"2022-06-06T00:00:00Z","timestamp":1654473600000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Front. Biosci. (Landmark Ed)"],"abstract":"<jats:p>Introduction: Spinal muscular atrophy (SMA) is a progressive neurological disease with autosomal recessive transmission that affects motor neurons, causing their loss and resulting in muscle waste and motor deficiency. Nusinersen, the first SMN2 pre-mRNA targeted therapy approved by the Food and Drug Administration and the European Medicines Agency, has demonstrated high efficacy in improving motor function, as well as respiratory and nutritional statuses. Materials and Methods: We observed 55 patients (children\/adolescents) diagnosed with spinal muscular atrophy (SMA), who received nusinersen therapy. To investigate the benefits of physical therapy on rehabilitation outcomes, we compared the motor evolution of patients who received nusinersen and performed daily physical therapy (study group) to those of the control group, who received only nusinersen therapy. Results: Motor skill improvements were statistically significantly (p &lt; 0.001) higher in the study group, being almost four times better (12.66%), effect size, in comparison to the control group (3.18%). Conclusions: Physical therapy has provided superior results for those who receive it on a regular basis. These results include the correction of posture, reduction in stiffness, expansion of the range of motion and strengthening of muscles, thus allowing patients to do more movements and boosting their ability to perform everyday tasks.<\/jats:p>","DOI":"10.31083\/j.fbl2706179","type":"journal-article","created":{"date-parts":[[2022,6,10]],"date-time":"2022-06-10T02:45:16Z","timestamp":1654829116000},"source":"Crossref","is-referenced-by-count":46,"title":["Physical Therapy and Nusinersen Impact on Spinal Muscular Atrophy Rehabilitative Outcome"],"prefix":"10.31083","volume":"27","author":[{"given":"Andrada","family":"Mirea","sequence":"first","affiliation":[{"name":"University of Medicine and Pharmacy \u201cCarol Davila\u201d, 020021 Bucharest, Romania"},{"name":"National Teaching Center for Children\u2019s Neurorehabilitation \u201cDr. Nicolae Robanescu\u201d, 041408 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Madalina Cristina","family":"Leanca","sequence":"first","affiliation":[{"name":"University of Medicine and Pharmacy \u201cCarol Davila\u201d, 020021 Bucharest, Romania"},{"name":"National Teaching Center for Children\u2019s Neurorehabilitation \u201cDr. Nicolae Robanescu\u201d, 041408 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gelu","family":"Onose","sequence":"first","affiliation":[{"name":"University of Medicine and Pharmacy \u201cCarol Davila\u201d, 020021 Bucharest, Romania"},{"name":"Teaching Emergency Hospital \u201cBagdasar-Arseni\u201d, 041915 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Corina","family":"Sporea","sequence":"first","affiliation":[{"name":"University of Medicine and Pharmacy \u201cCarol Davila\u201d, 020021 Bucharest, Romania"},{"name":"National Teaching Center for Children\u2019s Neurorehabilitation \u201cDr. Nicolae Robanescu\u201d, 041408 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liliana","family":"Padure","sequence":"first","affiliation":[{"name":"University of Medicine and Pharmacy \u201cCarol Davila\u201d, 020021 Bucharest, Romania"},{"name":"National Teaching Center for Children\u2019s Neurorehabilitation \u201cDr. Nicolae Robanescu\u201d, 041408 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena-Silvia","family":"Shelby","sequence":"first","affiliation":[{"name":"National Teaching Center for Children\u2019s Neurorehabilitation \u201cDr. Nicolae Robanescu\u201d, 041408 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vlad","family":"Dima","sequence":"first","affiliation":[{"name":"Clinical Hospital of Obstetrics and Gynecology \u201cFilantropia\u201d, 011132 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina","family":"Daia","sequence":"first","affiliation":[{"name":"University of Medicine and Pharmacy \u201cCarol Davila\u201d, 020021 Bucharest, Romania"},{"name":"Teaching Emergency Hospital \u201cBagdasar-Arseni\u201d, 041915 Bucharest, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"16242","published-online":{"date-parts":[[2022,6,6]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","unstructured":"Butchbach ME. Copy number variations in the survival motor neuron genes: implications for spinal muscular atrophy and other neurodegenerative diseases. Frontiers in Molecular Biosciences. 2016; 3: 7.","DOI":"10.3389\/fmolb.2016.00007"},{"key":"ref2","doi-asserted-by":"crossref","unstructured":"Wirth B. An update of the mutation spectrum of the survival motor neuron gene (SMN1) in autosomal recessive spinal muscular atrophy (SMA). Human Mutation. 2000; 15: 228\u2013237.","DOI":"10.1002\/(SICI)1098-1004(200003)15:3<228::AID-HUMU3>3.0.CO;2-9"},{"key":"ref3","doi-asserted-by":"crossref","unstructured":"Nash LA, Burns JK, Chardon JW, Kothary R, Parks RJ. Spinal Muscular Atrophy: more than a Disease of Motor Neurons? Current Molecular Medicine. 2016; 16: 779\u2013792.","DOI":"10.2174\/1566524016666161128113338"},{"key":"ref4","doi-asserted-by":"crossref","unstructured":"Scoto M, Finkel RS, Mercuri E, Muntoni F. Therapeutic approaches for spinal muscular atrophy (SMA). Gene Therapy. 2017; 24: 514\u2013519.","DOI":"10.1038\/gt.2017.45"},{"key":"ref5","doi-asserted-by":"crossref","unstructured":"Shelby ES, Nedelea FM, Huseyinoglu T, Coco\u0219 R, B\u0103dina M, Sporea C, et al. Innovative Therapies in Genetic Diseases: Cystic Fibrosis. Romanian Journal of Pediatrics. 2021; 70: 16\u201320.","DOI":"10.37897\/RJP.2021.1.3"},{"key":"ref6","doi-asserted-by":"crossref","unstructured":"Tisdale S, Pellizzoni L. Disease Mechanisms and Therapeutic Approaches in Spinal Muscular Atrophy. Journal of Neuroscience. 2015; 35: 8691\u20138700.","DOI":"10.1523\/JNEUROSCI.0417-15.2015"},{"key":"ref7","doi-asserted-by":"crossref","unstructured":"Kariya S, Park G, Maeno-Hikichi Y, Leykekhman O, Lutz C, Arkovitz MS, et al. Reduced SMN protein impairs maturation of the neuromuscular junctions in mouse models of spinal muscular atrophy. Human Molecular Genetics. 2008; 17: 2552\u20132569.","DOI":"10.1093\/hmg\/ddn156"},{"key":"ref8","doi-asserted-by":"crossref","unstructured":"Prior TW. Spinal muscular atrophy diagnostics. Journal of Child Neurology. 2007; 22: 952\u2013956.","DOI":"10.1177\/0883073807305668"},{"key":"ref9","doi-asserted-by":"crossref","unstructured":"He J, Zhang Q, Lin Q, Chen Y, Lin X, Lin M, et al. Molecular analysis of SMN1, SMN2, NAIP, GTF2H2, and H4F5 genes in 157 Chinese patients with spinal muscular atrophy. Gene. 2013; 518: 325\u2013329.","DOI":"10.1016\/j.gene.2012.12.109"},{"key":"ref10","doi-asserted-by":"crossref","unstructured":"Hoy SM. Nusinersen: first Global Approval. Drugs. 2017; 77: 473\u2013479.","DOI":"10.1007\/s40265-017-0711-7"},{"key":"ref11","doi-asserted-by":"crossref","unstructured":"Cartegni L, Hastings ML, Calarco JA, de Stanchina E, Krainer AR. Determinants of exon 7 splicing in the spinal muscular atrophy genes, SMN1 and SMN2. American Journal of Human Genetics. 2006; 78: 63\u201377.","DOI":"10.1086\/498853"},{"key":"ref12","doi-asserted-by":"crossref","unstructured":"Mailman MD, Heinz JW, Papp AC, Snyder PJ, Sedra MS, Wirth B, et al. Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2. Genetics in Medicine. 2002; 4: 20\u201326.","DOI":"10.1097\/00125817-200201000-00004"},{"key":"ref13","doi-asserted-by":"crossref","unstructured":"Wirth B, Brichta L, Schrank B, Lochm\u00fcller H, Blick S, Baasner A, et al. Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number. Human Genetics. 2006; 119: 422\u2013428.","DOI":"10.1007\/s00439-006-0156-7"},{"key":"ref14","doi-asserted-by":"crossref","unstructured":"Munsat TL, Davies KE. International SMA consortium meeting. Neuromuscular Disorders. 1992; 2: 423\u2013428.","DOI":"10.1016\/S0960-8966(06)80015-5"},{"key":"ref15","unstructured":"Types of SMA. 2021. Available at: https:\/\/rarediseases.org\/rare-diseases\/spinal-muscular-atrophy\/ (Accessed: 25 March 2022)."},{"key":"ref16","doi-asserted-by":"crossref","unstructured":"Dubowitz V. Very severe spinal muscular atrophy (SMA type 0): an expanding clinical phenotype. European Journal of Paediatric Neurology. 1999; 3: 49\u201351.","DOI":"10.1016\/S1090-3798(99)80001-4"},{"key":"ref17","unstructured":"Muscular Distrophy Association. 2022. Available at: https:\/\/www.mda.org\/disease\/spinal-muscular-atrophy\/types (Accessed: 1 February 2022)."},{"key":"ref18","doi-asserted-by":"crossref","unstructured":"Keinath MC, Prior DE, Prior TW. Spinal Muscular Atrophy: Mutations, Testing, and Clinical Relevance. The Application of Clinical Genetics. 2021; 14: 11\u201325.","DOI":"10.2147\/TACG.S239603"},{"key":"ref19","unstructured":"Spinal muscular atrophy. 2021. Available at: https:\/\/ghr.nlm.nih.gov\/condition\/spinal-muscular-atrophy#statistics (Accessed: 17 May 2021)."},{"key":"ref20","doi-asserted-by":"crossref","unstructured":"De Sanctis R, Coratti G, Pasternak A, Montes J, Pane M, Mazzone ES, et al. Developmental milestones in type i spinal muscular atrophy. Neuromuscular Disorders. 2016; 26: 754\u2013759.","DOI":"10.1016\/j.nmd.2016.10.002"},{"key":"ref21","doi-asserted-by":"crossref","unstructured":"Arnold WD, Kassar D, Kissel JT. Spinal muscular atrophy: diagnosis and management in a new therapeutic era. Muscle and Nerve. 2015; 51: 157\u2013167.","DOI":"10.1002\/mus.24497"},{"key":"ref22","doi-asserted-by":"crossref","unstructured":"Finkel RS, McDermott MP, Kaufmann P, Darras BT, Chung WK, Sproule DM, et al. Observational study of spinal muscular atrophy type i and implications for clinical trials. Neurology. 2014; 83: 810\u2013817.","DOI":"10.1212\/WNL.0000000000000741"},{"key":"ref23","doi-asserted-by":"crossref","unstructured":"Farrar MA, Kiernan MC. The Genetics of Spinal Muscular Atrophy: Progress and Challenges. Neurotherapeutics. 2015; 12: 290\u2013302.","DOI":"10.1007\/s13311-014-0314-x"},{"key":"ref24","doi-asserted-by":"crossref","unstructured":"Glascock J, Sampson J, Haidet-Phillips A, Connolly A, Darras B, Day J, et al. Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening. Journal of Neuromuscular Diseases. 2018; 5: 145\u2013158.","DOI":"10.3233\/JND-180304"},{"key":"ref25","doi-asserted-by":"crossref","unstructured":"Peredo DE, Hannibal MC. The Floppy Infant. Pediatrics in Review. 2009; 30: e66\u2013e76.","DOI":"10.1542\/pir.30.9.e66"},{"key":"ref26","doi-asserted-by":"crossref","unstructured":"Al-Zaidy SA, Mendell JR. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Pediatric Neurology. 2019; 100: 3\u201311.","DOI":"10.1016\/j.pediatrneurol.2019.06.007"},{"key":"ref27","doi-asserted-by":"crossref","unstructured":"Mercuri E, Finkel RS, Muntoni F, Wirth B, Montes J, Main M, et al. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscular Disorders. 2018; 28: 103\u2013115.","DOI":"10.1016\/j.nmd.2017.11.004"},{"key":"ref28","doi-asserted-by":"crossref","unstructured":"Finkel RS, Mercuri E, Meyer OH, Simonds AK, Schroth MK, Graham RJ, et al. Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. Neuromuscular Disorders. 2018; 28: 197\u2013207.","DOI":"10.1016\/j.nmd.2017.11.004"},{"key":"ref29","doi-asserted-by":"crossref","unstructured":"Chang RF, Mubarak SJ. Pathomechanics of Gowers\u2019 Sign: a Video Analysis of a Spectrum of Gowers\u2019 Maneuvers. Clinical Orthopaedics and Related Research. 2012; 470: 1987\u20131991.","DOI":"10.1007\/s11999-011-2210-6"},{"key":"ref30","unstructured":"U.S. Food & Drug Administration. 2022. Available at: https:\/\/www.fda.gov (Accessed: 22 March 2022)."},{"key":"ref31","unstructured":"European Medicines Agency. 2022. Available at: https:\/\/www.ema.europa.eu\/en (Accessed: 22 March 2022)."},{"key":"ref32","doi-asserted-by":"crossref","unstructured":"Gidaro T, Servais L. Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps. Developmental Medicine and Child Neurology. 2019; 61: 19\u201324.","DOI":"10.1111\/dmcn.14027"},{"key":"ref33","doi-asserted-by":"crossref","unstructured":"Baranello G, Darras BT, Day JW, Deconinck N, Klein A, Masson R, et al. Risdiplam in Type 1 Spinal Muscular Atrophy. New England Journal of Medicine. 2021; 384: 915\u2013923.","DOI":"10.1056\/NEJMoa2009965"},{"key":"ref34","doi-asserted-by":"crossref","unstructured":"Waldrop MA, Kolb SJ. Current Treatment Options in Neurology\u2014SMA Therapeutics. Current Treatment Options in Neurology. 2019; 21: 25.","DOI":"10.1007\/s11940-019-0568-z"},{"key":"ref35","unstructured":"Summarry of Product INN Nusinersen. 2017. Available at: https:\/\/www.ema.europa.eu\/en\/documents\/product-information\/spinraza-epar-product-information_en.pdf (Accessed: 2 February 2022)."},{"key":"ref36","unstructured":"AHFS approved Spinraza. 2016. Available at: https:\/\/www.ahfsdruginformation.com\/spinraza-nusinersen-approved\/ (Accessed: 24 February 2022)."},{"key":"ref37","unstructured":"EMA Nusinersen approved. 2018. Available at: https:\/\/www.ema.europa.eu\/en\/medicines\/human\/orphan-designations\/eu312976 (Accessed: 15 January 2022)."},{"key":"ref38","doi-asserted-by":"crossref","unstructured":"Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, et al. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes and Development. 2010; 24: 1634\u20131644.","DOI":"10.1101\/gad.1941310"},{"key":"ref39","unstructured":"Healthcare Information SAFETY - NUSINERSEN. 2021. Available at: https:\/\/www.spinraza.com\/en_us\/home\/taking-spinraza\/dosing.html (Accessed: 1 February 2022)."},{"key":"ref40","unstructured":"Intrathecal administration with SPINRAZA\u00ae (nusinersen). 2020. Available at: https:\/\/www.spinraza.com\/content\/dam\/commercial\/spinraza\/caregiver\/en_us\/pdf\/SPZ-US-0603-Intrathecal-Discussion-Tool.pdf (Accessed: 6 February 2022)."},{"key":"ref41","unstructured":"Figueiredo M. Motor Function Improved after Spinraza. 2020. Available at: https:\/\/smanewstoday.com\/2020\/06\/24\/spinraza-improves-motor-function-after-1-year-children-sma-type-1-2-real-life-study-france\/ (Accessed: 22 March 2022)."},{"key":"ref42","unstructured":"SMA News Today - Spinraza. 2021. Available at: https:\/\/smanewstoday.com\/spinraza-nusinersen\/ (Accessed: 4 February 2022)."},{"key":"ref43","doi-asserted-by":"crossref","unstructured":"LoMauro A, Mastella C, Alberti K, Masson R, Aliverti A, Baranello G. Effect of Nusinersen on Respiratory Muscle Function in Different Subtypes of Type 1 Spinal Muscular Atrophy. American Journal of Respiratory and Critical Care Medicine. 2019; 200: 1547\u20131550.","DOI":"10.1164\/rccm.201906-1175LE"},{"key":"ref44","doi-asserted-by":"crossref","unstructured":"Darras BT, Farrar MA, Mercuri E, Finkel RS, Foster R, Hughes SG, et al. An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials. CNS Drugs. 2019; 33: 919\u2013932.","DOI":"10.1007\/s40263-019-00656-w"},{"key":"ref45","doi-asserted-by":"crossref","unstructured":"Dangouloff T, Servais L. Clinical Evidence Supporting Early Treatment of Patients with Spinal Muscular Atrophy: Current Perspectives. Therapeutics and Clinical Risk Management. 2019; 15: 1153\u20131161.","DOI":"10.2147\/TCRM.S172291"},{"key":"ref46","doi-asserted-by":"crossref","unstructured":"De Vivo DC, Bertini E, Swoboda KJ, Hwu W, Crawford TO, Finkel RS, et al. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study. Neuromuscular Disorders. 2019; 29: 842\u2013856.","DOI":"10.1016\/j.nmd.2019.09.007"},{"key":"ref47","doi-asserted-by":"crossref","unstructured":"Strauss KA, Farrar MA, Swoboda KJ, Saito K, Chiriboga CA, Finkel RS, et al. Onasemnogene abeparvovec-xioi gene-replacement therapy in presymptomatic spinal muscular atrophy: SPR1NT study update (2384). AAN Enterprises. 2020.","DOI":"10.1212\/WNL.94.15_supplement.2384"},{"key":"ref48","doi-asserted-by":"crossref","unstructured":"Finkel RS, Al-Muhaizea M, Farrar MA, Nelson L, Prufer A, Servais L, et al. RAINBOWFISH: A Study of Risdiplam in Newborns with Presymptomatic Spinal Muscular Atrophy (SMA) (4281). AAN Enterprises. 2021.","DOI":"10.1212\/WNL.96.15_supplement.4281"},{"key":"ref49","doi-asserted-by":"crossref","unstructured":"Iannaccone ST. Modern management of spinal muscular atrophy. Journal of Child Neurology. 2007; 22: 974\u2013978.","DOI":"10.1177\/0883073807305670"},{"key":"ref50","doi-asserted-by":"crossref","unstructured":"Wang CH, Finkel RS, Bertini ES, Schroth M, Simonds A, Wong B, et al. Consensus statement for standard of care in spinal muscular atrophy. Journal of Child Neurology. 2007; 22: 1027\u20131049.","DOI":"10.1177\/0883073807305788"},{"key":"ref51","doi-asserted-by":"crossref","unstructured":"Dunaway S, Montes J, McDermott MP, Martens W, Neisen A, Glanzman AM, et al. Physical therapy services received by individuals with spinal muscular atrophy (SMA). Journal of Pediatric Rehabilitation Medicine. 2016; 9: 35\u201344.","DOI":"10.3233\/PRM-160360"},{"key":"ref52","doi-asserted-by":"crossref","unstructured":"St\u0119pie\u0144 A, J\u0119drzejowska M, Guzek K, Rekowski W, St\u0119powska J. Reliability of four tests to assess body posture and the range of selected movements in individuals with spinal muscular atrophy. BMC Musculoskeletal Disorders. 2019; 20: 54.","DOI":"10.1186\/s12891-018-2389-8"},{"key":"ref53","doi-asserted-by":"crossref","unstructured":"Onose G, Popescu N, Munteanu C, Ciobanu V, Sporea C, Mirea MD, et al. Mobile Mechatronic\/Robotic Orthotic Devices to Assist\u2013Rehabilitate Neuromotor Impairments in the Upper Limb: A Systematic and Synthetic Review. Frontiers in Neuroscience. 2018; 12: 577.","DOI":"10.3389\/fnins.2018.00577"},{"key":"ref54","doi-asserted-by":"crossref","unstructured":"Wang HY, Ju YH, Chen SM, Lo SK, Jong YJ. Joint range of motion limitations in children and young adults with spinal muscular atrophy. Archives of Physical Medicine and Rehabilitation. 2004; 85: 1689\u20131693.","DOI":"10.1016\/j.apmr.2004.01.043"},{"key":"ref55","doi-asserted-by":"crossref","unstructured":"Krosschell KJ, Maczulski JA, Crawford TO, Scott C, Swoboda KJ. A modified Hammersmith functional motor scale for use in multi-center research on spinal muscular atrophy. Neuromuscular Disorders. 2006; 16: 417\u2013426.","DOI":"10.1016\/j.nmd.2006.03.015"},{"key":"ref56","doi-asserted-by":"crossref","unstructured":"Main M, Kairon H, Mercuri E, Muntoni F. The Hammersmith functional motor scale for children with spinal muscular atrophy: a scale to test ability and monitor progress in children with limited ambulation. European Journal of Paediatric Neurology. 2003; 7: 155\u2013159.","DOI":"10.1016\/S1090-3798(03)00060-6"},{"key":"ref57","doi-asserted-by":"crossref","unstructured":"Bach JR, Vega J, Majors J, Friedman A. Spinal muscular atrophy type 1 quality of life. American Journal of Physical Medicine and Rehabilitation. 2003; 82: 137\u2013142.","DOI":"10.1097\/00002060-200302000-00009"},{"key":"ref58","doi-asserted-by":"crossref","unstructured":"Vaidya S, Boes S. Measuring quality of life in children with spinal muscular atrophy: a systematic literature review. Quality of Life Research. 2018; 27: 3087\u20133094.","DOI":"10.1007\/s11136-018-1945-x"},{"key":"ref59","doi-asserted-by":"crossref","unstructured":"de Oliveira CM, Ara\u00fajo APDQC. Self-reported quality of life has no correlation with functional status in children and adolescents with spinal muscular atrophy. European Journal of Paediatric Neurology. 2011; 15: 36\u201339.","DOI":"10.1016\/j.ejpn.2010.07.003"},{"key":"ref60","unstructured":"Mirea A, Padure L, Leanca MC, Marinescu GI, Preda B, Sporea C, et al. Kinesitherapy Impact On Rehabilitative Outcomes In Spinal Muscular Atrophy Nusinersen Treated Patients. Balneo Research Journal. 2020; 11: 98."},{"key":"ref61","doi-asserted-by":"crossref","unstructured":"Glanzman AM, Mazzone E, Main M, Pelliccioni M, Wood J, Swoboda KJ, et al. The Children\u2019s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): test development and reliability. Neuromuscular Disorders. 2010; 20: 155\u2013161.","DOI":"10.1016\/j.nmd.2009.11.014"},{"key":"ref62","doi-asserted-by":"crossref","unstructured":"Massey FJ. The Kolmogorov-Smirnov Test for Goodness of Fit. Journal of the American Statistical Association. 1951; 46: 68\u201378.","DOI":"10.1080\/01621459.1951.10500769"},{"key":"ref63","unstructured":"Pallant J. SPSS survival manual: a step by step guide to data analysis using IBM SPSS. 6th edn. McGraw-Hill Education: New York, NY, USA. 2016."},{"key":"ref64","unstructured":"White SE. Basic & Clinical Biostatistics. 5th edn. McGraw-Hill Education: New York, NY, USA. 2020."},{"key":"ref65","doi-asserted-by":"crossref","unstructured":"Kim TK. T test as a parametric statistic. Korean Journal of Anesthesiology. 2015; 68: 540\u2013546.","DOI":"10.4097\/kjae.2015.68.6.540"},{"key":"ref66","doi-asserted-by":"crossref","unstructured":"Ruxton GD. The unequal variance t-test is an underused alternative to Student\u2019s t-test and the Mann\u2013Whitney U test. Behavioral Ecology. 2006; 17: 688\u2013690.","DOI":"10.1093\/beheco\/ark016"},{"key":"ref67","doi-asserted-by":"crossref","unstructured":"Birnbaum ZW. On a use of the Mann-Whitney statistic. In Volume 1 Contribution to the Theory of Statistics (pp. 13\u201318). University of California Press: Berkeley, CA, USA. 2020.","DOI":"10.1525\/9780520313880-005"},{"key":"ref68","unstructured":"Lehmann EL, Romano JP, Casella G. Testing statistical hypotheses. vol. 3. Springer: New York. 2005."},{"key":"ref69","unstructured":"Armitage P, Berry G, Matthews JNS. Statistical methods in medical research. John Wiley & Sons: Hoboken, NJ, USA. 2008."},{"key":"ref70","doi-asserted-by":"crossref","unstructured":"Mix L, Winter B, Wurster CD, Platen S, Witzel S, Uzelac Z, et al. Quality of Life in SMA patients under treatment with nusinersen. Frontiers in Neurology. 2021; 12: 626787.","DOI":"10.3389\/fneur.2021.626787"},{"key":"ref71","doi-asserted-by":"crossref","unstructured":"Olsson B, Alberg L, Cullen NC, Michael E, Wahlgren L, Kroksmark AK, et al. NFL is a marker of treatment response in children with SMA treated with nusinersen. Journal of Neurology. 2019; 266: 2129\u20132136.","DOI":"10.1007\/s00415-019-09389-8"}],"container-title":["Frontiers in Bioscience-Landmark"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.imrpress.com\/journal\/FBL\/27\/6\/10.31083\/j.fbl2706179","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,11,27]],"date-time":"2024-11-27T11:55:14Z","timestamp":1732708514000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.imrpress.com\/journal\/FBL\/27\/6\/10.31083\/j.fbl2706179"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,6,6]]},"references-count":71,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2022]]}},"alternative-id":["S2768-6701(22)00540-8"],"URL":"https:\/\/doi.org\/10.31083\/j.fbl2706179","relation":{},"ISSN":["2768-6701","2768-6698"],"issn-type":[{"value":"2768-6701","type":"print"},{"value":"2768-6698","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,6,6]]},"article-number":"179"}}