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A survey of the feasibility of developing osteoporosis clinical trials in Duchenne muscular dystrophy: Survey of the opinion of young people with Duchenne muscular dystrophy, families and clinicians.
Choong Wong S, Joseph S, Capaldi N, Marco MD, Dunne J, Guglieri M, Horrocks I, Straub V, Faisal Ahmed S; UK NorthStar Clinical Network. Choong Wong S, et al. Among authors: horrocks i. Clin Trials. 2021 Feb;18(1):39-50. doi: 10.1177/1740774520958395. Epub 2020 Oct 4. Clin Trials. 2021. PMID: 33012180
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.
Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Hirtz D, Shieh PB, Straub V, Childs AM, Ciafaloni E, Butterfield RJ, Horrocks I, Spinty S, Flanigan KM, Kuntz NL, Baranello G, Roper H, Morrison L, Mah JK, Manzur AY, McDonald CM, Schara U, von der Hagen M, Barohn RJ, Campbell C, Darras BT, Finkel RS, Vita G, Hughes I, Mongini T, Pegoraro E, Wicklund M, Wilichowski E, Bryan Burnette W, Howard JF, McMillan HJ, Thangarajh M, Griggs RC. Guglieri M, et al. Among authors: horrocks i. Contemp Clin Trials. 2017 Jul;58:34-39. doi: 10.1016/j.cct.2017.04.008. Epub 2017 Apr 24. Contemp Clin Trials. 2017. PMID: 28450193 Free PMC article. Clinical Trial.
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.
Crow RA, Hart KA, McDermott MP, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Hirtz D, Lochmuller H, Straub V, Ciafaloni E, Shieh PB, Spinty S, Childs AM, Manzur AY, Morandi L, Butterfield RJ, Horrocks I, Roper H, Flanigan KM, Kuntz NL, Mah JK, Morrison L, Darras BT, von der Hagen M, Schara U, Wilichowski E, Mongini T, McDonald CM, Vita G, Barohn RJ, Finkel RS, Wicklund M, McMillan HJ Jr, Hughes I, Pegoraro E, Bryan Burnette W, Howard JF, Thangarajh M, Campbell C, Griggs RC, Bushby K, Guglieri M. Crow RA, et al. Among authors: horrocks i. Trials. 2018 May 10;19(1):291. doi: 10.1186/s13063-018-2645-0. Trials. 2018. PMID: 29793540 Free PMC article. Review.
Fractures and bone health monitoring in boys with Duchenne muscular dystrophy managed within the Scottish Muscle Network.
Joseph S, Wang C, Di Marco M, Horrocks I, Abu-Arafeh I, Baxter A, Cordeiro N, McLellan L, McWilliam K, Naismith K, Stephen E, Ahmed SF, Wong SC. Joseph S, et al. Among authors: horrocks i. Neuromuscul Disord. 2019 Jan;29(1):59-66. doi: 10.1016/j.nmd.2018.09.005. Epub 2018 Sep 25. Neuromuscul Disord. 2019. PMID: 30473133 Free article.
Fractures and Linear Growth in a Nationwide Cohort of Boys With Duchenne Muscular Dystrophy With and Without Glucocorticoid Treatment: Results From the UK NorthStar Database.
Joseph S, Wang C, Bushby K, Guglieri M, Horrocks I, Straub V, Ahmed SF, Wong SC; UK NorthStar Clinical Network. Joseph S, et al. Among authors: horrocks i. JAMA Neurol. 2019 Jun 1;76(6):701-709. doi: 10.1001/jamaneurol.2019.0242. JAMA Neurol. 2019. PMID: 30855644 Free PMC article.
45 results