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In this study, we investigate median change in the RHS score up to two years in paediatric SMA 2 and 3 participants and contextualise it to the Hammersmith Functional Motor Scale\u2013Expanded (HFMSE). These change scores were considered by SMA type, motor function, and baseline RHS score. We consider a new transitional group, spanning crawlers, standers, and walkers-with-assistance, and analyse that alongside non-sitters, sitters, and walkers. The transitional group exhibit the most definitive change score trend, with an average 1-year decline of 3 points. In the weakest patients, we are most able to detect positive change in the RHS in the under-5 age group, whereas in the stronger patients, we are most able to detect decline in the RHS in the 8\u201313 age group. The RHS has a reduced floor effect compared to the HFMSE, although we show that the RHS should be used in conjunction with the RULM for participants scoring less than 20 points on the RHS. The timed items in the RHS have high between-participant variability, so participants with the same RHS total can be differentiated by their timed test items.<\/jats:p>","DOI":"10.3390\/jcm12051920","type":"journal-article","created":{"date-parts":[[2023,3,1]],"date-time":"2023-03-01T01:36:09Z","timestamp":1677634569000},"page":"1920","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":13,"title":["2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA Participants"],"prefix":"10.3390","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2795-9898","authenticated-orcid":false,"given":"Georgia","family":"Stimpson","sequence":"first","affiliation":[{"name":"Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Danielle","family":"Ramsey","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK"},{"name":"School of Health and Sports Sciences, University of Suffolk, Ipswich IP4 1QJ, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy","family":"Wolfe","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Mayhew","sequence":"additional","affiliation":[{"name":"The John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle NE1 7RU, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariacristina","family":"Scoto","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4871-6692","authenticated-orcid":false,"given":"Giovanni","family":"Baranello","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK"},{"name":"National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London WC1N 1EH, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8901-9140","authenticated-orcid":false,"given":"Robert","family":"Muni Lofra","sequence":"additional","affiliation":[{"name":"The John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle NE1 7RU, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marion","family":"Main","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7129-1824","authenticated-orcid":false,"given":"Evelin","family":"Milev","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6666-5628","authenticated-orcid":false,"given":"Giorgia","family":"Coratti","sequence":"additional","affiliation":[{"name":"Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy"},{"name":"Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marika","family":"Pane","sequence":"additional","affiliation":[{"name":"Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy"},{"name":"Centro Clinico Nemo, U.O.C. 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Neurol."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1212\/WNL.0000000000006502","article-title":"Evidence in Focus: Nusinersen Use in Spinal Muscular Atrophy Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology","volume":"91","author":"Michelson","year":"2018","journal-title":"Neurology"},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"779","DOI":"10.1001\/archneurol.2010.373","article-title":"Observational Study of Spinal Muscular Atrophy Type 2 and 3: Functional Outcomes over 1 Year","volume":"68","author":"Kaufmann","year":"2011","journal-title":"Arch. Neurol."},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1016\/j.ncl.2015.07.004","article-title":"Spinal Muscular Atrophy","volume":"33","author":"Kolb","year":"2015","journal-title":"Neurol. Clin."},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"3017","DOI":"10.1016\/S0140-6736(16)31408-8","article-title":"Treatment of Infantile-Onset Spinal Muscular Atrophy with Nusinersen: A Phase 2, Open-Label, Dose-Escalation Study","volume":"388","author":"Finkel","year":"2016","journal-title":"Lancet"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"e2492","DOI":"10.1212\/WNL.0000000000007527","article-title":"Nusinersen in Later-Onset Spinal Muscular Atrophy: Long-Term Results from the Phase 1\/2 Studies","volume":"92","author":"Darras","year":"2019","journal-title":"Neurology"},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1007\/s40263-019-00656-w","article-title":"An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials","volume":"33","author":"Darras","year":"2019","journal-title":"CNS Drugs"},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"1853","DOI":"10.1007\/s40265-020-01410-z","article-title":"Risdiplam: First Approval","volume":"80","author":"Dhillon","year":"2020","journal-title":"Drugs"},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"689","DOI":"10.1038\/s41372-021-01301-5","article-title":"Is Treatment with Oral Risdiplam Effective and Well-Tolerated for Infants with Spinal Muscular Atrophy Type 1?","volume":"42","author":"Gaffar","year":"2022","journal-title":"J. Perinatol."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1056\/NEJMoa2102047","article-title":"Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls","volume":"385","author":"Darras","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"693","DOI":"10.1016\/j.nmd.2007.05.009","article-title":"An Expanded Version of the Hammersmith Functional Motor Scale for SMA II and III Patients","volume":"17","author":"Glanzman","year":"2007","journal-title":"Neuromuscul. Disord."},{"key":"ref_13","doi-asserted-by":"crossref","unstructured":"Ramsey, D., Scoto, M., Mayhew, A., Main, M., Mazzone, E.S., Montes, J., de Sanctis, R., Young, S.D., Salazar, R., and Glanzman, A.M. (2017). Revised Hammersmith Scale for Spinal Muscular Atrophy: A SMA Specific Clinical Outcome Assessment Tool. PLoS ONE, 12.","DOI":"10.1371\/journal.pone.0172346"},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"101","DOI":"10.1002\/pri.520","article-title":"Development of a Functional Assessment Scale for Ambulatory Boys with Duchenne Muscular Dystrophy","volume":"17","author":"Scott","year":"2012","journal-title":"Physiother. Res. Int."},{"key":"ref_15","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":"Glanzman","year":"2011","journal-title":"J. Child Neurol."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"794","DOI":"10.1016\/j.nmd.2019.08.014","article-title":"Development of an Academic Disease Registry for Spinal Muscular Atrophy","volume":"29","author":"Mercuri","year":"2019","journal-title":"Neuromuscul. Disord."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"1027","DOI":"10.1177\/0883073807305788","article-title":"Consensus Statement for Standard of Care in Spinal Muscular Atrophy Current Problems in the Medical Care of Patients With Spinal Muscular Atrophy Spinal Muscular Atrophy Is a Recessively Inherited Neuro-Muscular Disease Characterized by Degeneration of Spinal","volume":"22","author":"Wang","year":"2007","journal-title":"J. Child Neurol."},{"key":"ref_18","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":"Mercuri","year":"2018","journal-title":"Neuromuscul. Disord."},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"197","DOI":"10.1016\/j.nmd.2017.11.004","article-title":"Diagnosis and Management of Spinal Muscular Atrophy: Part 2: Pulmonary and Acute Care; Medications, Supplements and Immunizations; Other Organ Systems; and Ethics","volume":"28","author":"Finkel","year":"2018","journal-title":"Neuromuscul. Disord."},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"756","DOI":"10.1016\/j.nmd.2020.07.005","article-title":"Age and Baseline Values Predict 12 and 24-Month Functional Changes in Type 2 SMA","volume":"30","author":"Coratti","year":"2020","journal-title":"Neuromuscul. Disord."},{"key":"ref_21","unstructured":"Ramsey, D., Mayhew, A., Scoto, M., Baranello, G., Stimpson, G., Muni Lofra, R., Main, M., Montes, J., Glanzman, A., and Pasternak, A. (2022, January 21\u201323). Using Functional Phenotypes and Not Type to Chart Physical Ability in SMA 2 & 3. Proceedings of the 3rd International Scientific Congress on Spinal Muscular Atrophy, Barcelona, Spain."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1016\/j.nmd.2015.10.006","article-title":"Patterns of Disease Progression in Type 2 and 3 SMA: Implications for Clinical Trials","volume":"26","author":"Mercuri","year":"2016","journal-title":"Neuromuscul. Disord."},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"404","DOI":"10.1002\/acn3.51514","article-title":"Nusinersen Efficacy Data for 24-month in Type 2 and 3 Spinal Muscular Atrophy","volume":"9","author":"Pane","year":"2022","journal-title":"Ann. Clin. Transl. Neurol."},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"552","DOI":"10.1002\/mus.27384","article-title":"Different Trajectories in Upper Limb and Gross Motor Function in Spinal Muscular Atrophy","volume":"64","author":"Coratti","year":"2021","journal-title":"Muscle Nerve"},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"1109","DOI":"10.1002\/ana.25900","article-title":"Clinical Variability in Spinal Muscular Atrophy Type III","volume":"88","author":"Coratti","year":"2020","journal-title":"Ann. Neurol."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1016\/j.nmd.2020.07.004","article-title":"Gain and Loss of Abilities in Type II SMA: A 12-Month Natural History Study","volume":"30","author":"Coratti","year":"2020","journal-title":"Neuromuscul. Disord."},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"596","DOI":"10.1016\/j.nmd.2021.03.012","article-title":"Age Related Treatment Effect in Type II Spinal Muscular Atrophy Pediatric Patients Treated with Nusinersen","volume":"31","author":"Coratti","year":"2021","journal-title":"Neuromuscul. Disord."},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"1622","DOI":"10.1002\/acn3.51411","article-title":"Nusinersen in Pediatric and Adult Patients with Type III Spinal Muscular Atrophy","volume":"8","author":"Pera","year":"2021","journal-title":"Ann. Clin. Transl. 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