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Page 1
Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy.
Ayyar Gupta V, Pitchforth JM, Domingos J, Ridout D, Iodice M, Rye C, Chesshyre M, Wolfe A, Selby V, Mayhew A, Mazzone ES, Ricotti V, Hogrel JY, Niks EH, de Groot I, Servais L, Straub V, Mercuri E, Manzur AY, Muntoni F; iMDEX Consortium and the U.K. NorthStar Clinical Network. Ayyar Gupta V, et al. Among authors: hogrel jy. PLoS One. 2023 Apr 26;18(4):e0283669. doi: 10.1371/journal.pone.0283669. eCollection 2023. PLoS One. 2023. PMID: 37099511 Free PMC article.
Effect of sirolimus on muscle in inclusion body myositis observed with magnetic resonance imaging and spectroscopy.
Reyngoudt H, Baudin PY, de Caldas de Almeida Araújo E, Bachasson D, Boisserie JM, Mariampillai K, Annoussamy M, Allenbach Y, Hogrel JY, Carlier PG, Marty B, Benveniste O. Reyngoudt H, et al. Among authors: hogrel jy. J Cachexia Sarcopenia Muscle. 2024 Apr 13. doi: 10.1002/jcsm.13451. Online ahead of print. J Cachexia Sarcopenia Muscle. 2024. PMID: 38613252 Free article.
A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice.
Woodcock IR, Tachas G, Desem N, Houweling PJ, Kean M, Emmanuel J, Kennedy R, Carroll K, de Valle K, Adams J, Lamandé SR, Coles C, Tiong C, Burton M, Villano D, Button P, Hogrel JY, Catling-Seyffer S, Ryan MM, Delatycki MB, Yiu EM. Woodcock IR, et al. Among authors: hogrel jy. PLoS One. 2024 Jan 25;19(1):e0294847. doi: 10.1371/journal.pone.0294847. eCollection 2024. PLoS One. 2024. PMID: 38271438 Free PMC article. Clinical Trial.
Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2-3 trial.
Köhler W, Engelen M, Eichler F, Lachmann R, Fatemi A, Sampson J, Salsano E, Gamez J, Molnar MJ, Pascual S, Rovira M, Vilà A, Pina G, Martín-Ugarte I, Mantilla A, Pizcueta P, Rodríguez-Pascau L, Traver E, Vilalta A, Pascual M, Martinell M, Meya U, Mochel F; ADVANCE Study Group. Köhler W, et al. Lancet Neurol. 2023 Feb;22(2):127-136. doi: 10.1016/S1474-4422(22)00495-1. Lancet Neurol. 2023. PMID: 36681445 Clinical Trial.
Three-year quantitative magnetic resonance imaging and phosphorus magnetic resonance spectroscopy study in lower limb muscle in dysferlinopathy.
Reyngoudt H, Smith FE, Caldas de Almeida Araújo E, Wilson I, Fernández-Torrón R, James MK, Moore UR, Díaz-Manera J, Marty B, Azzabou N, Gordish H, Rufibach L, Hodgson T, Wallace D, Ward L, Boisserie JM, Le Louër J, Hilsden H, Sutherland H, Canal A, Hogrel JY, Jacobs M, Stojkovic T, Bushby K, Mayhew A; Jain Clinical Outcome Study for Dysferlinopathy consortium; Straub V, Carlier PG, Blamire AM. Reyngoudt H, et al. Among authors: hogrel jy. J Cachexia Sarcopenia Muscle. 2022 Jun;13(3):1850-1863. doi: 10.1002/jcsm.12987. Epub 2022 Apr 3. J Cachexia Sarcopenia Muscle. 2022. PMID: 35373496 Free PMC article.
Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.
Mayhew AG, James MK, Moore U, Sutherland H, Jacobs M, Feng J, Lowes LP, Alfano LN, Muni Lofra R, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sánchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Gordish-Dressman H, Hilsden H, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Straub V. Mayhew AG, et al. Among authors: hogrel jy. Front Neurol. 2022 Mar 10;13:828525. doi: 10.3389/fneur.2022.828525. eCollection 2022. Front Neurol. 2022. PMID: 35359643 Free PMC article.
Genotype-related respiratory progression in Duchenne muscular dystrophy-A multicenter international study.
Trucco F, Ridout D, Domingos J, Maresh K, Chesshyre M, Munot P, Sarkozy A, Robb S, Quinlivan R, Riley M, Wallis C, Chan E, Abel F, De Lucia S, Hogrel JY, Niks EH, de Groot I, Servais L, Straub V, Ricotti V, Manzur A, Muntoni F; UK NorthStar Clinical Network and AFM Network. Trucco F, et al. Among authors: hogrel jy. Muscle Nerve. 2022 Jan;65(1):67-74. doi: 10.1002/mus.27427. Epub 2021 Oct 27. Muscle Nerve. 2022. PMID: 34606104
189 results