{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,28]],"date-time":"2026-06-28T03:46:07Z","timestamp":1782618367097,"version":"3.54.5"},"reference-count":16,"publisher":"SAGE Publications","issue":"4","license":[{"start":{"date-parts":[[2025,9,23]],"date-time":"2025-09-23T00:00:00Z","timestamp":1758585600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2025,9,23]],"date-time":"2025-09-23T00:00:00Z","timestamp":1758585600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2026,7]]},"abstract":"<jats:p>We described ambulatory Duchenne muscular dystrophy (DMD) progression, across multiple functional measures, via previously established prognostic groups for loss of ambulation (LoA) and health states. Patients closer to vs. farther from LoA had greater declines in some measures (e.g., 6-min walk distance) and less change in others (e.g., timed rise from floor velocity) due to floor effects. Patients in the late vs. early ambulatory health state were concordantly shifted towards higher LoA risk. Findings further characterize health states and prognostic factors in ambulatory DMD and highlight the importance of multiple measures of function to fully characterize disease progression.<\/jats:p>","DOI":"10.1177\/22143602251364694","type":"journal-article","created":{"date-parts":[[2025,9,23]],"date-time":"2025-09-23T15:15:22Z","timestamp":1758640522000},"page":"781-787","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Descriptive characterization of ambulatory health states in Duchenne muscular dystrophy: Motor function trajectories and times to loss of ambulation"],"prefix":"10.1177","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9102-5232","authenticated-orcid":false,"given":"Francesco","family":"Muntoni","sequence":"first","affiliation":[{"name":"Dubowitz Neuromuscular Centre, Great Ormond Street Institute of Child Health, London, UK"},{"name":"NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James","family":"Signorovitch","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA, USA"},{"name":"Collaborative Trajectory Analysis Project (cTAP), Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michaela","family":"Johnson","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8320-0857","authenticated-orcid":false,"given":"Andres","family":"Gomez-Lievano","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nate","family":"Posner","sequence":"additional","affiliation":[{"name":"Pfizer, Inc., New York, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patricia","family":"Dorling","sequence":"additional","affiliation":[{"name":"Pfizer, Inc., New York, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katherine","family":"Beaverson","sequence":"additional","affiliation":[{"name":"Pfizer, Inc., New York, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jose","family":"Alvir","sequence":"additional","affiliation":[{"name":"Pfizer, Inc., New York, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1629-9372","authenticated-orcid":false,"given":"Matthias","family":"Mahn","sequence":"additional","affiliation":[{"name":"Pfizer, Inc., New York, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan J","family":"Ward","sequence":"additional","affiliation":[{"name":"Collaborative Trajectory Analysis Project (cTAP), Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathalie","family":"Goemans","sequence":"additional","affiliation":[{"name":"Child Neurology, University Hospitals Leuven, Leuven, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Krista","family":"Vandenborne","sequence":"additional","affiliation":[{"name":"University of Florida, Gainesville, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9851-5365","authenticated-orcid":false,"given":"Eugenio","family":"Mercuri","sequence":"additional","affiliation":[{"name":"Pediatric Neurology, Catholic University, Rome, Italy"},{"name":"Centro Clinico NeMO, Fondazione Policlinico Gemelli IRCCS, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig M","family":"McDonald","sequence":"additional","affiliation":[{"name":"Department of Physical Medicine and Rehabilitation, University of California, Sacramento, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2025,9,23]]},"reference":[{"key":"e_1_3_3_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"e_1_3_3_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(19)32910-1"},{"key":"e_1_3_3_4_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2016.05.016"},{"key":"e_1_3_3_5_2","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.15176"},{"key":"e_1_3_3_6_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002891"},{"key":"e_1_3_3_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)32160-8"},{"key":"e_1_3_3_8_2","volume-title":"A prognostic score for time to loss of ambulation in Duchenne muscular dystrophyPoster 68 presented at the 27th International Hybrid Annual Congress of the World Muscle Society","author":"McDonald C","year":"2022","unstructured":"McDonald C, Gordish-Dressman H, Signorovitch J, et al. A prognostic score for time to loss of ambulation in Duchenne muscular dystrophy. In: Poster 68 presented at the 27th International Hybrid Annual Congress of the World Muscle Society, Halifax, Canada, October 11-15, 2022. https:\/\/ctap-duchenne.org\/wp-content\/uploads\/2023\/12\/2022_WMS_Poster_A-Prognostic-Score-for-Time-to-Loss-of-Ambulation.pdf."},{"key":"e_1_3_3_9_2","doi-asserted-by":"publisher","DOI":"10.1007\/s41669-023-00450-x"},{"key":"e_1_3_3_10_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0307118"},{"key":"e_1_3_3_11_2","doi-asserted-by":"publisher","DOI":"10.1002\/pri.520"},{"key":"e_1_3_3_12_2","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2024-335223"},{"key":"e_1_3_3_13_2","volume-title":"Functional trajectories of upper limb and pulmonary function before and after loss of ambulation in Duchenne muscular dystrophyPoster 103 presented at the Muscular Dystrophy Association Conference","author":"Goemans N","year":"2021","unstructured":"Goemans N, Signorovitch J, McDonald C, et al. Functional trajectories of upper limb and pulmonary function before and after loss of ambulation in Duchenne muscular dystrophy. In: Poster 103 presented at the Muscular Dystrophy Association Conference, Virtual, March 15-18, 2021."},{"key":"e_1_3_3_14_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2023.07.073"},{"key":"e_1_3_3_15_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23902"},{"key":"e_1_3_3_16_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000201626"},{"key":"e_1_3_3_17_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0304099"}],"container-title":["Journal of Neuromuscular Diseases"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/22143602251364694","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/22143602251364694","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/22143602251364694","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,6,28]],"date-time":"2026-06-28T03:33:23Z","timestamp":1782617603000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/22143602251364694"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,9,23]]},"references-count":16,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2026,7]]}},"alternative-id":["10.1177\/22143602251364694"],"URL":"https:\/\/doi.org\/10.1177\/22143602251364694","relation":{},"ISSN":["2214-3599","2214-3602"],"issn-type":[{"value":"2214-3599","type":"print"},{"value":"2214-3602","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,9,23]]}}}