Publications du réseau

Compte tenu de son activité, les publications du réseau portent sur des analyses et des réflexions destinées aux acteurs et aux décideurs.

  • Cortial L, Montero V, Tourlet S, Del Bano J, Blin O. Artificial intelligence in drug repurposing for rare diseases: a mini-review. Front Med (Lausanne). 2024 May 22;11:1404338. doi: 10.3389/fmed.2024.1404338. 
  • Lutsyk K, Gicquel T, Cortial L, Forget S, Braun S, Boyer PO, Laugel V, Blin O. Does gene therapies clinical research in rare diseases reflects the competitivity of the country: Example of France. Therapie. 2024 Feb 15:S0040-5957(24)00028-3. doi: 10.1016/j.therap.2024.01.007. 
  • Cortial L, Nguyen C, Julkowska D, Cocqueel-Tiran F, Moliner AM, Blin O, Trentesaux V. Managing rare diseases: examples of national approaches in Europe, North America and East Asia. Rare Dis Orphan Drugs J 2022;1:10. doi:10.20517/rdodj.2022.02 
  • Cortial L, Boyer PO, Forget S, Le Joubioux R, Blin O, Mouthon F. Rare diseases: specific challenges for sustainable accessibility of treatments for patients. Rare Dis Orphan Drugs J 2022;1:12. doi:10.20517/rdodj.2022.08 
  • Gicquel T, Cortial L, Lutsyk K, Forget S, Braun S, Boyer PO, Laugel V, Blin O. 2017-2023: state of the art of gene therapies in rare diseases in Europe: the dynamics of clinical R&D, new approved treatments and expected therapies in the pipelines. Rare Dis Orphan Drugs J 2023;2:23. doi:10.20517/rdodj.2023.29 
  • Blin O, Micallef J. Drugs and orphan diseases. Therapie. 2020 Apr;75(2):131-132. doi: 10.1016/j.therap.2020.02.002. 
  • Blin O, Lefebvre MN, Rascol O, Micallef J. Orphan drug clinical development. Therapie. 2020 Apr;75(2):141-147. doi: 10.1016/j.therap.2020.02.004. 
  • Micallef J, Blin O. Orphan drug designation in Europe: A booster for the research and development of drugs in rare diseases. Therapie. 2020 Apr;75(2):133-139. doi: 10.1016/j.therap.2020.02.003. 
  • Micallef J, Boutouyrie P, Blin O. Pharmacology and drug development in rare diseases: the attractiveness and expertise of the French medical pharmacology. Fundam Clin Pharmacol. 2017 Dec;31(6):685-694. doi: 10.1111/fcp.12314. 
  • Colomban C, Micallef J, Lefebvre MN, Dubourg O, Gonnaud PM, Stojkovic T, Jouve E, Blin O, Pouget J, Attarian S. Clinical spectrum and gender differences in a large cohort of Charcot-Marie-Tooth type 1A patients. J Neurol Sci. 2014 Jan 15;336(1-2):155-60. doi: 10.1016/j.jns.2013.10.029. Epub 2013 Oct 29. PMID: 24246498.

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Mis à jour le 23 juillet 2024