PMID- 37804000 OWN - NLM STAT- MEDLINE DCOM- 20231216 LR - 20240221 IS - 2328-9503 (Electronic) IS - 2328-9503 (Linking) VI - 10 IP - 12 DP - 2023 Dec TI - Association between neurodevelopmental impairments and motor function in Duchenne muscular dystrophy. PG - 2285-2296 LID - 10.1002/acn3.51914 [doi] AB - OBJECTIVE: We explored various prognostic factors of motor outcomes in corticosteroid-naive boys with Duchenne Muscular Dystrophy (DMD). METHODS: The associations between parent-reported neurodevelopmental concerns (speech delay, speech and language difficulties (SLD), and learning difficulties), DMD mutation location, and motor outcomes (6-minute walk distance (6MWD), North Star Ambulatory Assessment (NSAA) total score, 10-meter walk/run velocity, and rise from floor velocity) were studied in 196 corticosteroid-naive boys from ages 4 to less than 8 years. RESULTS: Participants with SLD walked 25.8 fewer meters in 6 minutes than those without SLD (p = 0.005) but did not demonstrate statistical differences in NSAA total score, 10-meter walk/run velocity, and rise from floor velocity. Participants with distal DMD mutations with learning difficulties walked 51.8 fewer meters in 6 minutes than those without learning difficulties (p = 0.0007). Participants with distal DMD mutations were slower on 10-meter walk/run velocity, and rise from floor velocity (p = 0.02) than those with proximal DMD mutations. Participants with distal DMD mutations, who reported speech delay or learning difficulties, were slower on rise from floor velocity (p = 0.04, p = 0.01) than those with proximal DMD mutations. The mean NSAA total score was lower in participants with learning difficulties than in those without (p = 0.004). INTERPRETATION: Corticosteroid-naive boys with DMD with distal DMD mutations may perform worse on some timed function tests, and that those with learning difficulties may perform worse on the NSAA. Pending confirmatory studies, our data underscore the importance of considering co-existing neurodevelopmental symptoms on motor outcome measures. CI - (c) 2023 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association. FAU - Thangarajh, Mathula AU - Thangarajh M AUID- ORCID: 0000-0001-9811-0838 AD - Department of Neurology, Virginia Commonwealth University, Richmond, Virginia, USA. FAU - McDermott, Michael P AU - McDermott MP AD - University of Rochester Medical Center, School of Medicine and Dentistry, Rochester, New York, USA. FAU - Guglieri, Michela AU - Guglieri M AD - John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals National Health Service Foundation Trust, Newcastle, UK. FAU - Griggs, Robert C AU - Griggs RC AD - University of Rochester Medical Center, School of Medicine and Dentistry, Rochester, New York, USA. LA - eng GR - R21 TR004007/TR/NCATS NIH HHS/United States GR - U01 NS061795/NS/NINDS NIH HHS/United States GR - U01 NS061799/NS/NINDS NIH HHS/United States GR - UM1 TR004360/TR/NCATS NIH HHS/United States GR - R21TR004007/TR/NCATS NIH HHS/United States GR - K12 TR004364/TR/NCATS NIH HHS/United States PT - Journal Article PT - Research Support, N.I.H., Extramural PT - Research Support, Non-U.S. Gov't DEP - 20231007 PL - United States TA - Ann Clin Transl Neurol JT - Annals of clinical and translational neurology JID - 101623278 RN - 0 (Adrenal Cortex Hormones) SB - IM MH - Male MH - Humans MH - *Muscular Dystrophy, Duchenne/complications/genetics MH - Walking MH - Adrenal Cortex Hormones MH - Outcome Assessment, Health Care MH - *Language Development Disorders PMC - PMC10723228 COIS- Dr Guglieri reported receiving grants from Duchenne UK, the European Union's Horizon 2020 program for the Vision-DMD study (in collaboration with ReveraGen BioPharma Inc), and Sarepta Therapeutics; serving as a consultant to Dyne Therapeutics Inc, Pfizer, and NS Pharma Inc; receiving personal fees from Sarepta Therapeutics; and receiving nonfinancial support from Italfarmaco, Pfizer, ReveraGen BioPharma Inc, and Santhera Pharmaceuticals. Dr McDermott reported receiving grant support from PTC Therapeutics and receiving personal fees from Fulcrum Therapeutics, NeuroDerm Ltd, AstraZeneca, Eli Lilly, Catabasis Pharmaceuticals, Vaccinex Inc, Cynapsus Therapeutics, Neurocrine Biosciences, Voyager Therapeutics, Prilenia Therapeutics, ReveraGen BioPharma Inc, and NS Pharma Inc. Dr Thangarajh reported serving as a consultant to Sarepta Therapeutics and speaker for NS Pharma Inc. Dr Griggs reported serving as a consultant to Strongbridge and Stealth Pharmaceuticals; receiving personal fees from Solid Biosciences and Elsevier; serving as chair of the research advisory committee and is a board member of the American Brain Foundation; and serving on the executive committee of the Muscle Study Group, which receives support for its activities from pharmaceutical companies. EDAT- 2023/10/07 11:43 MHDA- 2023/12/17 09:43 PMCR- 2023/10/07 CRDT- 2023/10/07 03:34 PHST- 2023/07/13 00:00 [revised] PHST- 2023/05/22 00:00 [received] PHST- 2023/09/09 00:00 [accepted] PHST- 2023/12/17 09:43 [medline] PHST- 2023/10/07 11:43 [pubmed] PHST- 2023/10/07 03:34 [entrez] PHST- 2023/10/07 00:00 [pmc-release] AID - ACN351914 [pii] AID - 10.1002/acn3.51914 [doi] PST - ppublish SO - Ann Clin Transl Neurol. 2023 Dec;10(12):2285-2296. doi: 10.1002/acn3.51914. Epub 2023 Oct 7.