{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T20:03:07Z","timestamp":1785614587246,"version":"3.56.0"},"reference-count":31,"publisher":"Public Library of Science (PLoS)","issue":"3","license":[{"start":{"date-parts":[[2024,3,19]],"date-time":"2024-03-19T00:00:00Z","timestamp":1710806400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100008792","name":"Novartis Pharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008792","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"abstract":"<jats:sec id=\"sec001\">\n<jats:title>Purpose<\/jats:title>\n<jats:p>This single-arm prospective cohort study aimed to evaluate the feasibility and utility of in-home body weight support harness system (BWSS) use in children treated for spinal muscular atrophy (SMA).<\/jats:p>\n<\/jats:sec>\n<jats:sec id=\"sec002\">\n<jats:title>Methods<\/jats:title>\n<jats:p>Individuals with 2 or 3 copies of <jats:italic>SMN2<\/jats:italic> who received pharmacotherapeutic treatment, had head control, and weight &lt;50lbs were enrolled. Families were provided a BWSS and documented use. Motor outcome assessments were completed at baseline, month 3 and month 6. Families provided feedback in an end of study survey.<\/jats:p>\n<\/jats:sec>\n<jats:sec id=\"sec003\">\n<jats:title>Results<\/jats:title>\n<jats:p>All 32 participants (2.9 (SD 1.9) yrs), improved or remained stable on all outcomes. Average reported frequency of use was 4.1(2.3) hrs\/week. Controlling for other covariates, frequency of use explained over 70% of the variability in change scores. Family feedback was overwhelmingly positive.<\/jats:p>\n<\/jats:sec>\n<jats:sec id=\"sec004\">\n<jats:title>Conclusion<\/jats:title>\n<jats:p>Use of in-home BWSS is a safe, feasible and useful option to increase exercise dosage after treatment in SMA and may help optimize motor abilities.<\/jats:p>\n<\/jats:sec>\n<jats:sec id=\"sec005\">\n<jats:title>Trial registration<\/jats:title>\n<jats:p>Study registered with: Clinicaltrials.gov<\/jats:p>\n<jats:p>Clinicaltrials.gov identifier: <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT05715749\" xlink:type=\"simple\">NCT05715749<\/jats:ext-link>.<\/jats:p>\n<\/jats:sec>","DOI":"10.1371\/journal.pone.0300244","type":"journal-article","created":{"date-parts":[[2024,3,19]],"date-time":"2024-03-19T17:24:27Z","timestamp":1710869067000},"page":"e0300244","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":5,"title":["Feasibility and utility of in-home body weight support harness system use in young children treated for spinal muscular atrophy: A single-arm prospective cohort study"],"prefix":"10.1371","volume":"19","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2800-3855","authenticated-orcid":true,"given":"Megan A.","family":"Iammarino","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lindsay N.","family":"Alfano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalie F.","family":"Reash","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brenna","family":"Sabo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sara","family":"Conroy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7308-3810","authenticated-orcid":true,"given":"Garey","family":"Noritz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3897-1315","authenticated-orcid":true,"given":"Madalynn","family":"Wendland","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda P.","family":"Lowes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2024,3,19]]},"reference":[{"issue":"2","key":"pone.0300244.ref001","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1016\/j.nmd.2017.11.005","article-title":"Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care","volume":"28","author":"E Mercuri","year":"2018","journal-title":"Neuromuscular disorders: NMD"},{"issue":"5\u20136","key":"pone.0300244.ref002","doi-asserted-by":"crossref","first-page":"423","DOI":"10.1016\/S0960-8966(06)80015-5","article-title":"International SMA consortium meeting. (26\u201328 June 1992, Bonn, Germany)","volume":"2","author":"TL Munsat","year":"1992","journal-title":"Neuromuscular disorders: NMD"},{"issue":"8","key":"pone.0300244.ref003","doi-asserted-by":"crossref","first-page":"1027","DOI":"10.1177\/0883073807305788","article-title":"Consensus statement for standard of care in spinal muscular atrophy","volume":"22","author":"CH Wang","year":"2007","journal-title":"J Child Neurol"},{"issue":"4","key":"pone.0300244.ref004","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1016\/j.ncl.2015.07.004","article-title":"Spinal Muscular Atrophy","volume":"33","author":"SJ Kolb","year":"2015","journal-title":"Neurologic clinics"},{"issue":"18","key":"pone.0300244.ref005","doi-asserted-by":"crossref","first-page":"1723","DOI":"10.1056\/NEJMoa1702752","article-title":"Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy","volume":"377","author":"RS Finkel","year":"2017","journal-title":"The New England journal of medicine"},{"issue":"18","key":"pone.0300244.ref006","doi-asserted-by":"crossref","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","article-title":"Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy","volume":"377","author":"JR Mendell","year":"2017","journal-title":"The New England journal of medicine"},{"issue":"10","key":"pone.0300244.ref007","doi-asserted-by":"crossref","first-page":"915","DOI":"10.1056\/NEJMoa2009965","article-title":"Risdiplam in Type 1 Spinal Muscular Atrophy","volume":"384","author":"G Baranello","year":"2021","journal-title":"The New England journal of medicine"},{"key":"pone.0300244.ref008","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1016\/j.pediatrneurol.2019.05.005","article-title":"Impact of Age and Motor Function in a Phase 1\/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy","volume":"98","author":"LP Lowes","year":"2019","journal-title":"Pediatr Neurol"},{"issue":"11","key":"pone.0300244.ref009","doi-asserted-by":"crossref","first-page":"842","DOI":"10.1016\/j.nmd.2019.09.007","article-title":"Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study","volume":"29","author":"DC De Vivo","year":"2019","journal-title":"Neuromuscular disorders: NMD"},{"issue":"3","key":"pone.0300244.ref010","doi-asserted-by":"crossref","DOI":"10.1542\/peds.2020-0729","article-title":"Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes","volume":"146","author":"MA Waldrop","year":"2020","journal-title":"Pediatrics"},{"issue":"1","key":"pone.0300244.ref011","first-page":"35","article-title":"Physical therapy services received by individuals with spinal muscular atrophy (SMA)","volume":"9","author":"S Dunaway","year":"2016","journal-title":"J Pediatr Rehabil Med"},{"issue":"2","key":"pone.0300244.ref012","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1002\/mus.24497","article-title":"Spinal muscular atrophy: diagnosis and management in a new therapeutic era","volume":"51","author":"WD Arnold","year":"2015","journal-title":"Muscle Nerve"},{"issue":"11","key":"pone.0300244.ref013","first-page":"Cd013114","article-title":"Mechanically assisted walking training for walking, participation, and quality of life in children with cerebral palsy","volume":"11","author":"HC Chiu","year":"2020","journal-title":"Cochrane Database Syst Rev"},{"issue":"1","key":"pone.0300244.ref014","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1097\/NPT.0b013e31819800e2","article-title":"A systematic review of the effectiveness of treadmill training and body weight support in pediatric rehabilitation","volume":"33","author":"DL Damiano","year":"2009","journal-title":"J Neurol Phys Ther"},{"issue":"3","key":"pone.0300244.ref015","doi-asserted-by":"crossref","first-page":"E1","DOI":"10.1097\/PEP.0000000000000516","article-title":"Use of an In-Home Body Weight Support System by a Child With Spina Bifida","volume":"30","author":"E Kokkoni","year":"2018","journal-title":"Pediatr Phys Ther"},{"issue":"11","key":"pone.0300244.ref016","doi-asserted-by":"crossref","first-page":"1387","DOI":"10.1177\/0956797612446346","article-title":"How do you learn to walk? Thousands of steps and dozens of falls per day","volume":"23","author":"KE Adolph","year":"2012","journal-title":"Psychol Sci"},{"issue":"8","key":"pone.0300244.ref017","first-page":"Cd002840","article-title":"Treadmill training and body weight support for walking after stroke","volume":"8","author":"J Mehrholz","year":"2017","journal-title":"Cochrane Database Syst Rev"},{"key":"pone.0300244.ref018","doi-asserted-by":"crossref","first-page":"i5239","DOI":"10.1136\/bmj.i5239","article-title":"CONSORT 2010 statement: extension to randomised pilot and feasibility trials","volume":"355","author":"SM Eldridge","year":"2016","journal-title":"BMJ"},{"key":"pone.0300244.ref019","volume-title":"University CS","author":"A RD Sabet","year":"2017"},{"key":"pone.0300244.ref020","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1016\/j.pediatrneurol.2021.05.021","article-title":"Validity and Reliability of the Neuromuscular Gross Motor Outcome","volume":"122","author":"LN Alfano","year":"2021","journal-title":"Pediatr Neurol"},{"issue":"12","key":"pone.0300244.ref021","doi-asserted-by":"crossref","first-page":"1499","DOI":"10.1177\/0883073811420294","article-title":"Validation of the Expanded Hammersmith Functional Motor Scale in spinal muscular atrophy type II and III","volume":"26","author":"AM Glanzman","year":"2011","journal-title":"J Child Neurol"},{"issue":"2","key":"pone.0300244.ref022","doi-asserted-by":"crossref","first-page":"e0172346","DOI":"10.1371\/journal.pone.0172346","article-title":"Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool","volume":"12","author":"D Ramsey","year":"2017","journal-title":"PLoS One"},{"key":"pone.0300244.ref023","article-title":"Bayley scales of infant and toddler development: Bayley-III: Harcourt Assessment","author":"N Bayley","year":"2006","journal-title":"San Antonio: PsychCorp"},{"issue":"1 Suppl","key":"pone.0300244.ref024","doi-asserted-by":"crossref","first-page":"S37","DOI":"10.1177\/15648265040251S106","article-title":"Assessment of gross motor development in the WHO Multicentre Growth Reference Study","volume":"25","author":"TM Wijnhoven","year":"2004","journal-title":"Food Nutr Bull"},{"issue":"2","key":"pone.0300244.ref025","doi-asserted-by":"crossref","first-page":"246","DOI":"10.1002\/mus.22040","article-title":"Reliability of the Modified Hammersmith Functional Motor Scale in young children with spinal muscular atrophy","volume":"44","author":"KJ Krosschell","year":"2011","journal-title":"Muscle Nerve"},{"issue":"5","key":"pone.0300244.ref026","doi-asserted-by":"crossref","first-page":"913s","DOI":"10.1093\/ajcn\/79.5.913S","article-title":"The evolution of physical activity recommendations: how much is enough?","volume":"79","author":"SN Blair","year":"2004","journal-title":"Am J Clin Nutr"},{"key":"pone.0300244.ref027","first-page":"1","article-title":"RESNA position on the application of power mobility devices for pediatric users","author":"L Rosen","year":"2017","journal-title":"Assist Technol"},{"key":"pone.0300244.ref028","doi-asserted-by":"crossref","first-page":"299","DOI":"10.3389\/fped.2017.00299","article-title":"Modified Ride-On Cars and Young Children with Disabilities: Effects of Combining Mobility and Social Training","volume":"5","author":"HH Huang","year":"2017","journal-title":"Front Pediatr"},{"issue":"3","key":"pone.0300244.ref029","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1097\/PEP.0000000000000713","article-title":"Stander Use in Spinal Muscular Atrophy: Results From a Large Natural History Database","volume":"32","author":"EL Townsend","year":"2020","journal-title":"Pediatr Phys Ther"},{"issue":"3","key":"pone.0300244.ref030","doi-asserted-by":"crossref","first-page":"248","DOI":"10.1097\/PEP.0b013e318299127d","article-title":"Effects of passive versus dynamic loading interventions on bone health in children who are nonambulatory","volume":"25","author":"M Damcott","year":"2013","journal-title":"Pediatr Phys Ther"},{"issue":"3","key":"pone.0300244.ref031","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1097\/PEP.0b013e318299d5e7","article-title":"Systematic review and evidence-based clinical recommendations for dosing of pediatric supported standing programs","volume":"25","author":"GS Paleg","year":"2013","journal-title":"Pediatr Phys Ther"}],"container-title":["PLOS ONE"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pone.0300244","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,3,19]],"date-time":"2024-03-19T17:24:49Z","timestamp":1710869089000},"score":1,"resource":{"primary":{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pone.0300244"}},"subtitle":[],"editor":[{"given":"Andrea","family":"Martinuzzi","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"editor"}]}],"short-title":[],"issued":{"date-parts":[[2024,3,19]]},"references-count":31,"journal-issue":{"issue":"3","published-online":{"date-parts":[[2024,3,19]]}},"URL":"https:\/\/doi.org\/10.1371\/journal.pone.0300244","relation":{},"ISSN":["1932-6203"],"issn-type":[{"value":"1932-6203","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,3,19]]}}}