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Page 1
Propelling Healthcare with Advanced Therapy Medicinal Products: A Policy Discussion.
Horgan D, Metspalu A, Ouillade MC, Athanasiou D, Pasi J, Adjali O, Harrison P, Hermans C, Codacci-Pisanelli G, Koeva J, Szucs T, Cursaru V, Belina I, Bernini C, Zhuang S, McMahon S, Toncheva D, Thum T. Horgan D, et al. Among authors: ouillade mc. Biomed Hub. 2020 Dec 3;5(3):130-152. doi: 10.1159/000511678. eCollection 2020 Sep-Dec. Biomed Hub. 2020. PMID: 33987187 Free PMC article.
Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational.
McCormack P, Woods S, Aartsma-Rus A, Hagger L, Herczegfalvi A, Heslop E, Irwin J, Kirschner J, Moeschen P, Muntoni F, Ouillade MC, Rahbek J, Rehmann-Sutter C, Rouault F, Sejersen T, Vroom E, Straub V, Bushby K, Ferlini A. McCormack P, et al. Among authors: ouillade mc. PLoS Curr. 2013 Jan 10;5:ecurrents.md.f90b49429fa814bd26c5b22b13d773ec. doi: 10.1371/currents.md.f90b49429fa814bd26c5b22b13d773ec. PLoS Curr. 2013. PMID: 23330068 Free PMC article.
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.
Position Statement: Sharing of Clinical Research Data in Spinal Muscular Atrophy to Accelerate Research and Improve Outcomes for Patients.
Lochmüller H, Evans D, Farwell W, Finkel R, Goemans N, de Lemus M, Matyushenko V, Muntoni F, Ouillade MC, Schwersenz I, Wilson P. Lochmüller H, et al. Among authors: ouillade mc. J Neuromuscul Dis. 2018;5(2):131-133. doi: 10.3233/JND-180325. J Neuromuscul Dis. 2018. PMID: 29865093 Free PMC article. No abstract available.
[The expert patient: a new key stakeholder in the global healthcare system].
Friconneau M, Archer A, Malaterre J, Salama F, Ouillade MC. Friconneau M, et al. Among authors: ouillade mc. Med Sci (Paris). 2020 Dec;36 Hors série n° 2:62-64. doi: 10.1051/medsci/2020206. Epub 2021 Jan 11. Med Sci (Paris). 2020. PMID: 33427642 Free article. Review. French.
270th ENMC International Workshop: Consensus for SMN2 genetic analysis in SMA patients 10-12 March, 2023, Hoofddorp, the Netherlands.
Abiusi E, Costa-Roger M, Bertini ES, Tiziano FD, Tizzano EF; SMN2 Study group; Abiusi E, Baranello G, Bertini E, Boemer F, Burghes A, Codina-Solà M, Costa-Roger M, Dangouloff T, Groen E, Gos M, Jędrzejowska M, Kirschner J, Lemmink HH, Müller-Felber W, Ouillade MC, Quijano-Roy S, Rucinski K, Saugier-Veber P, Tiziano FD, Tizzano EF, Wirth B. Abiusi E, et al. Among authors: ouillade mc. Neuromuscul Disord. 2024 Jan;34:114-122. doi: 10.1016/j.nmd.2023.12.008. Epub 2023 Dec 14. Neuromuscul Disord. 2024. PMID: 38183850 Free article.