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A decade of optimizing drug development for rare neuromuscular disorders through TACT.
Wagner KR, De Luca A, Caizergues D, Dowling J, Goemans N, Gordish-Dressman H, Grounds MD, Kelly M, Mayhew A, McNally EM, Zoetis T, Lee J, Turner C, Wells DJ, Csimma C, Straub V. Wagner KR, et al. Among authors: de luca a. Nat Rev Drug Discov. 2020 Jan;19(1):1-2. doi: 10.1038/d41573-019-00199-1. Nat Rev Drug Discov. 2020. PMID: 31907422 No abstract available.
Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy.
Lynn S, Aartsma-Rus A, Bushby K, Furlong P, Goemans N, De Luca A, Mayhew A, McDonald C, Mercuri E, Muntoni F, Pohlschmidt M, Verschuuren J, Voit T, Vroom E, Wells DJ, Straub V. Lynn S, et al. Among authors: de luca a. Neuromuscul Disord. 2015 Jan;25(1):96-105. doi: 10.1016/j.nmd.2014.09.003. Epub 2014 Sep 11. Neuromuscul Disord. 2015. PMID: 25307856 No abstract available.
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development.
Heslop E, Csimma C, Straub V, McCall J, Nagaraju K, Wagner KR, Caizergues D, Korinthenberg R, Flanigan KM, Kaufmann P, McNeil E, Mendell J, Hesterlee S, Wells DJ, Bushby K; TACT. Heslop E, et al. Orphanet J Rare Dis. 2015 Apr 23;10:49. doi: 10.1186/s13023-015-0258-1. Orphanet J Rare Dis. 2015. PMID: 25902795 Free PMC article. Review.
Update on Standard Operating Procedures in Preclinical Research for DMD and SMA Report of TREAT-NMD Alliance Workshop, Schiphol Airport, 26 April 2015, The Netherlands.
van Putten M, Aartsma-Rus A, Grounds MD, Kornegay JN, Mayhew A, Gillingwater TH, Takeda S, Rüegg MA, De Luca A, Nagaraju K, Willmann R. van Putten M, et al. Among authors: de luca a. J Neuromuscul Dis. 2018;5(1):29-34. doi: 10.3233/JND-170288. J Neuromuscul Dis. 2018. PMID: 29480217 Free PMC article.
Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.
Straub V, Balabanov P, Bushby K, Ensini M, Goemans N, De Luca A, Pereda A, Hemmings R, Campion G, Kaye E, Arechavala-Gomeza V, Goyenvalle A, Niks E, Veldhuizen O, Furlong P, Stoyanova-Beninska V, Wood MJ, Johnson A, Mercuri E, Muntoni F, Sepodes B, Haas M, Vroom E, Aartsma-Rus A. Straub V, et al. Among authors: de luca a. Lancet Neurol. 2016 Jul;15(8):882-890. doi: 10.1016/S1474-4422(16)30035-7. Lancet Neurol. 2016. PMID: 27302365 Review.
"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic.
Gordish-Dressman H, Willmann R, Dalle Pazze L, Kreibich A, van Putten M, Heydemann A, Bogdanik L, Lutz C, Davies K, Demonbreun AR, Duan D, Elsey D, Fukada SI, Girgenrath M, Patrick Gonzalez J, Grounds MD, Nichols A, Partridge T, Passini M, Sanarica F, Schnell FJ, Wells DJ, Yokota T, Young CS, Zhong Z, Spurney C, Spencer M, De Luca A, Nagaraju K, Aartsma-Rus A. Gordish-Dressman H, et al. Among authors: de luca a. J Neuromuscul Dis. 2018;5(4):407-417. doi: 10.3233/JND-180324. J Neuromuscul Dis. 2018. PMID: 30198876 Free PMC article.
2,256 results