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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: vroom e. 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.
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: vroom e. Lancet Neurol. 2016 Jul;15(8):882-890. doi: 10.1016/S1474-4422(16)30035-7. Lancet Neurol. 2016. PMID: 27302365 Review.
Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues.
Aartsma-Rus A, Straub V, Hemmings R, Haas M, Schlosser-Weber G, Stoyanova-Beninska V, Mercuri E, Muntoni F, Sepodes B, Vroom E, Balabanov P. Aartsma-Rus A, et al. Among authors: vroom e. Nucleic Acid Ther. 2017 Oct;27(5):251-259. doi: 10.1089/nat.2017.0682. Epub 2017 Aug 10. Nucleic Acid Ther. 2017. PMID: 28796573 Free PMC article. Review.
226th ENMC International Workshop:: Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy 20-22 January 2017, Heemskerk, The Netherlands.
Aartsma-Rus A, Ferlini A, McNally EM, Spitali P, Sweeney HL; workshop participants. Aartsma-Rus A, et al. Neuromuscul Disord. 2018 Jan;28(1):77-86. doi: 10.1016/j.nmd.2017.10.002. Epub 2017 Oct 26. Neuromuscul Disord. 2018. PMID: 29203356 Free PMC article. No abstract available.
Nutrition in Duchenne muscular dystrophy 16-18 March 2018, Zaandam, the Netherlands.
Verhaart IEC, van den Engel-Hoek L, Fiorotto ML, Franken-Verbeek M, Vroom E; workshop participants. Verhaart IEC, et al. Among authors: vroom e. Neuromuscul Disord. 2018 Aug;28(8):680-689. doi: 10.1016/j.nmd.2018.05.004. Epub 2018 May 16. Neuromuscul Disord. 2018. PMID: 29910096 No abstract available.
A Transition Toolkit for Duchenne Muscular Dystrophy.
Trout CJ, Case LE, Clemens PR, McArthur A, Noritz G, Ritzo M, Wagner KR, Vroom E, Kennedy A. Trout CJ, et al. Among authors: vroom e. Pediatrics. 2018 Oct;142(Suppl 2):S110-S117. doi: 10.1542/peds.2018-0333M. Pediatrics. 2018. PMID: 30275255
46 results