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18 results

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Page 1
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.
McDonald CM, Marbán E, Hendrix S, Hogan N, Ruckdeschel Smith R, Eagle M, Finkel RS, Tian C, Janas J, Harmelink MM, Varadhachary AS, Taylor MD, Hor KN, Mayer OH, Henricson EK, Furlong P, Ascheim DD, Rogy S, Williams P, Marbán L; HOPE-2 Study Group. McDonald CM, et al. Lancet. 2022 Mar 12;399(10329):1049-1058. doi: 10.1016/S0140-6736(22)00012-5. Lancet. 2022. PMID: 35279258 Free article. Clinical Trial.
Efficacy and Safety of Bimagrumab in Sporadic Inclusion Body Myositis: Long-term Extension of RESILIENT.
Amato AA, Hanna MG, Machado PM, Badrising UA, Chinoy H, Benveniste O, Karanam AK, Wu M, Tankó LB, Schubert-Tennigkeit AA, Papanicolaou DA, Lloyd TE, Needham M, Liang C, Reardon KA, de Visser M, Ascherman DP, Barohn RJ, Dimachkie MM, Miller JAL, Kissel JT, Oskarsson B, Joyce NC, Van den Bergh P, Baets J, De Bleecker JL, Karam C, David WS, Mirabella M, Nations SP, Jung HH, Pegoraro E, Maggi L, Rodolico C, Filosto M, Shaibani AI, Sivakumar K, Goyal NA, Mori-Yoshimura M, Yamashita S, Suzuki N, Aoki M, Katsuno M, Morihata H, Murata K, Nodera H, Nishino I, Romano CD, Williams VSL, Vissing J, Zhang Auberson L; RESILIENT Study Extension Group. Amato AA, et al. Neurology. 2021 Mar 23;96(12):e1595-e1607. doi: 10.1212/WNL.0000000000011626. Epub 2021 Feb 17. Neurology. 2021. PMID: 33597289 Free PMC article. Clinical Trial.
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy.
Wolff JM, Capocci N, Atas E, Bharucha-Goebel DX, Brandsema JF, Butterfield RJ, Chadwick CB, Corti M, Crawford TO, Cripe L, Day JW, Duong T, ElMallah MK, Flanigan KM, George LA, Goedeker NL, Goude E, Hesterlee S, Lin B, Katz NK, Matesanz SE, McDonald C, McNally EM, Mercado-Rodriguez C, Nandi D, Parsons JA, Proud C, Ramos-Platt L, Lek A, Salabarria SM, Camino E, Schrader R, Shea E, Shell R, Shieh PB, Soslow JH, Taylor JB, Veerapandiyan A, Villa C, Yang ML, Zaidman CM, Leon-Astudillo C, Byrne BJ. Wolff JM, et al. Among authors: goude e. Neuromuscul Disord. 2025 Sep;54:106208. doi: 10.1016/j.nmd.2025.106208. Epub 2025 Aug 23. Neuromuscul Disord. 2025. PMID: 41005046 Review.
ENLITE PD: A Randomized Clinical Trial of Light Therapy for Impaired Sleep in Parkinson's Disease.
Videnovic A, Coffey CS, Klerman EB, Cudkowicz M, Cho HJ, Amara A, Marder K, Zee P, Desir C, Ernst H, Steinhart E, Costigan M, Gudjonsdottir A, Klingner E, Ecklund D, Chase M, Wang A, Charles D, Clifford D, Coleman J, Espay AJ, Hung AY, Kadimi S, Killoran A, Luo L, Malkani R, Margolesky J, Marsella J, Maurer C, Mihaila D, Moretti P, Park A, Phielipp N, Sarva H, Shah B, Shill HA, Siderowf A, Suski V, Tate J, Tse W, Wyant K, Zhang L, Annis C, Ambrogi K, Auerbach G, Benge M, Blystone E, Brenner L, Aguilar CB, Bwala G, Dorsch J, Goude E, Gutierrez H, Harper K, Hochberger S, Kalyani P, Lamb J, Maupin K, Molloy A, Mosovsky S, Sissine M, Steele J, Szela M, Teshome M, Wencel M, Macklin EA; NeuroNEXT NN110 ENLITE PD Investigators. Videnovic A, et al. Among authors: goude e. Mov Disord. 2025 Nov;40(11):2445-2456. doi: 10.1002/mds.70009. Epub 2025 Sep 6. Mov Disord. 2025. PMID: 40913446 Clinical Trial.
Relationships between DMD mutations and neurodevelopment in dystrophinopathy.
Thangarajh M, Hendriksen J, McDermott MP, Martens W, Hart KA, Griggs RC; Muscle Study Group and TREAT-NMD. Thangarajh M, et al. Neurology. 2019 Oct 22;93(17):e1597-e1604. doi: 10.1212/WNL.0000000000008363. Epub 2019 Oct 8. Neurology. 2019. PMID: 31594858 Free PMC article. Clinical Trial.
Ataluren treatment of patients with nonsense mutation dystrophinopathy.
Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP, Connolly AM, Day JW, Flanigan KM, Goemans N, Jones KJ, Mercuri E, Quinlivan R, Renfroe JB, Russman B, Ryan MM, Tulinius M, Voit T, Moore SA, Lee Sweeney H, Abresch RT, Coleman KL, Eagle M, Florence J, Gappmaier E, Glanzman AM, Henricson E, Barth J, Elfring GL, Reha A, Spiegel RJ, O'donnell MW, Peltz SW, Mcdonald CM; PTC124-GD-007-DMD STUDY GROUP. Bushby K, et al. Muscle Nerve. 2014 Oct;50(4):477-87. doi: 10.1002/mus.24332. Muscle Nerve. 2014. PMID: 25042182 Free PMC article. Clinical Trial.
Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy.
Connolly AM, Malkus EC, Mendell JR, Flanigan KM, Miller JP, Schierbecker JR, Siener CA, Golumbek PT, Zaidman CM, Mcdonald CM, Johnson L, Nicorici A, Karachunski PI, Day JW, Kelecic JM, Lowes LP, Alfano LN, Darras BT, Kang PB, Quigley J, Pasternak AE, Florence JM; MDA DMD Clinical Research Network. Connolly AM, et al. Muscle Nerve. 2015 Apr;51(4):522-32. doi: 10.1002/mus.24346. Epub 2015 Feb 11. Muscle Nerve. 2015. PMID: 25056178 Free PMC article.
(-)-Epicatechin induces mitochondrial biogenesis and markers of muscle regeneration in adults with Becker muscular dystrophy.
McDonald CM, Ramirez-Sanchez I, Oskarsson B, Joyce N, Aguilar C, Nicorici A, Dayan J, Goude E, Abresch RT, Villarreal F, Ceballos G, Perkins G, Dugar S, Schreiner G, Henricson EK. McDonald CM, et al. Among authors: goude e. Muscle Nerve. 2021 Feb;63(2):239-249. doi: 10.1002/mus.27108. Epub 2020 Dec 16. Muscle Nerve. 2021. PMID: 33125736 Free PMC article.
Gait Characterization in Duchenne Muscular Dystrophy (DMD) Using a Single-Sensor Accelerometer: Classical Machine Learning and Deep Learning Approaches.
Ramli AA, Liu X, Berndt K, Goude E, Hou J, Kaethler LB, Liu R, Lopez A, Nicorici A, Owens C, Rodriguez D, Wang J, Zhang H, Aranki D, McDonald CM, Henricson EK. Ramli AA, et al. Among authors: goude e. Sensors (Basel). 2024 Feb 8;24(4):1123. doi: 10.3390/s24041123. Sensors (Basel). 2024. PMID: 38400281 Free PMC article.
18 results