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Page 1
Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies.
Fischer R, Furlong P, Kennedy A, Maynard K, Penrod M, Miller D, Laverty CG, Lowes LP, Kuntz NL, Shieh PB, Kondejewski J, Neumann PJ, Shafrin J, Willke RJ. Fischer R, et al. Among authors: laverty cg. J Multidiscip Healthc. 2024 Aug 29;17:4199-4212. doi: 10.2147/JMDH.S458181. eCollection 2024. J Multidiscip Healthc. 2024. PMID: 39224484 Free PMC article.
Missense variants in CMS22 patients reveal that PREPL has both enzymatic and nonenzymatic functions.
Monnens Y, Theodoropoulou A, Rosier K, Bhalla K, Mahy A, Vanhoutte R, Meulemans S, Cavani E, Antanasijevic A, Lemmens I, Lee JA, Spellicy CJ, Schroer RJ, Maselli RA, Laverty CG, Agostinis P, Pagliarini DJ, Verhelst S, Marcaida MJ, Rochtus A, Dal Peraro M, Creemers JW. Monnens Y, et al. Among authors: laverty cg. JCI Insight. 2024 Sep 10;9(17):e179276. doi: 10.1172/jci.insight.179276. JCI Insight. 2024. PMID: 39078710 Free PMC article.
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Mercuri E, Vilchez JJ, Boespflug-Tanguy O, Zaidman CM, Mah JK, Goemans N, Müller-Felber W, Niks EH, Schara-Schmidt U, Bertini E, Comi GP, Mathews KD, Servais L, Vandenborne K, Johannsen J, Messina S, Spinty S, McAdam L, Selby K, Byrne B, Laverty CG, Carroll K, Zardi G, Cazzaniga S, Coceani N, Bettica P, McDonald CM; EPIDYS Study Group. Mercuri E, et al. Among authors: laverty cg. Lancet Neurol. 2024 Apr;23(4):393-403. doi: 10.1016/S1474-4422(24)00036-X. Lancet Neurol. 2024. PMID: 38508835 Clinical Trial.
Clinical, immunohistochemical, and genetic characterization of splice-altering biallelic DES variants: Therapeutic implications.
Geist Hauserman J, Laverty CG, Donkervoort S, Hu Y, Silverstein S, Neuhaus SB, Saade D, Vaughn G, Malicki D, Kaur R, Li Y, Luo Y, Liu P, Burr P, Foley AR, Mohassel P, Bönnemann CG. Geist Hauserman J, et al. Among authors: laverty cg. HGG Adv. 2024 Apr 11;5(2):100274. doi: 10.1016/j.xhgg.2024.100274. Epub 2024 Feb 2. HGG Adv. 2024. PMID: 38358893 Free PMC article.