{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,21]],"date-time":"2026-08-21T12:45:46Z","timestamp":1787316346414,"version":"build-2736575974"},"reference-count":19,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2018,8,28]],"date-time":"2018-08-28T00:00:00Z","timestamp":1535414400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100007013","name":"F. Hoffmann-La Roche","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007013","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2018,10]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>\n                    <jats:italic>Introduction<\/jats:italic>\n                    : We propose a mathematical model to empirically describe spinal muscular atrophy (SMA) progression assessed by the 3 domains of the motor function measure (MFM) scale. The model implements development and deterioration of muscle function.\n                    <jats:italic>Methods<\/jats:italic>\n                    : Nonlinear mixed\u2010effects modeling was applied to data from 2 observational studies and 1 prospective clinical efficacy study comprising 190 healthy participants and 277 patients with type 2\/3 SMA.\n                    <jats:italic>Results<\/jats:italic>\n                    : The model evidenced correlations between parameter estimates for different MFM domains. Slower development in MFM domain D1 (standing and transfers) was associated with faster deterioration for MFM domains D2 (proximal and axial motricity) and D3 (distal motor function).\n                    <jats:italic>Discussion<\/jats:italic>\n                    : The model describes all individual data well, although sparseness and variability of observational data prevented numerically stable estimation of parameters. Treatment duration in clinical studies was too limited to determine a proper drug\u2010effect model that could differentiate between symptomatic and disease modifying effects.\n                    <jats:italic>Muscle Nerve<\/jats:italic>\n                    <jats:bold>58<\/jats:bold>\n                    : 528\u2013535, 2018\n                  <\/jats:p>","DOI":"10.1002\/mus.26178","type":"journal-article","created":{"date-parts":[[2018,8,9]],"date-time":"2018-08-09T07:49:16Z","timestamp":1533800956000},"page":"528-535","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Mathematical Disease Progression Modeling in Type 2\/3 Spinal Muscular Atrophy"],"prefix":"10.1002","volume":"58","author":[{"given":"Philippe","family":"Jacqmin","sequence":"first","affiliation":[{"name":"SGS\u2010Exprimo  Mechelen Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ronald","family":"Gieschke","sequence":"additional","affiliation":[{"name":"F. 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Hoffmann\u2013La Roche, Roche Innovation Center Basel  Grenzacher Street 124, Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carole","family":"Vuillerot","sequence":"additional","affiliation":[{"name":"Hospices Civils de Lyon, H\u00f4pital Femme\u2010M\u00e8re\u2010Enfant, L'Escale, Service de M\u00e9decine Physique et de R\u00e9adaptation P\u00e9diatrique  Bron France"},{"name":"Universit\u00e9 de Lyon  Lyon France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2904-5857","authenticated-orcid":false,"given":"Patricia","family":"Sanwald Ducray","sequence":"additional","affiliation":[{"name":"F. 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