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Improving outcome measures in late onset Pompe disease: Modified Rasch-Built Pompe-Specific Activity scale.
van Kooten HA, Horton MC, Wenninger S, Babačić H, Schoser B, Lefeuvre C, Taouagh N, Laforêt P, Segovia S, Díaz-Manera J, Claeys KG, Mongini T, Musumeci O, Toscano A, Hundsberger T, Brusse E, van Doorn PA, van der Ploeg AT, van der Beek NAME; European Pompe Consortium study group on outcome measures. van Kooten HA, et al. Eur J Neurol. 2024 Aug 28:e16397. doi: 10.1111/ene.16397. Online ahead of print. Eur J Neurol. 2024. PMID: 39205420
Effect of sirolimus on muscle in inclusion body myositis observed with magnetic resonance imaging and spectroscopy.
Reyngoudt H, Baudin PY, 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 Jun;15(3):1108-1120. doi: 10.1002/jcsm.13451. Epub 2024 Apr 13. J Cachexia Sarcopenia Muscle. 2024. PMID: 38613252 Free PMC article. Clinical Trial.
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.
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.
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.
192 results