Patient‐driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis

@article{Monaco2017PatientdrivenSF,
  title={Patient‐driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis},
  author={Lucia Monaco and Lucia Faccio},
  journal={EMBO Molecular Medicine},
  year={2017},
  volume={9},
  pages={289 - 292}
}
The recent approval of Strimvelis, the first ex vivo gene therapy to gain marketing authorization (Schimmer & Breazzano, ), has drawn attention to Fondazione Telethon, the Italian charity that played a pivotal role in this effort. Although it is not uncommon that advanced therapies, such as Strimvelis, are developed by partnerships between academia and industry, direct involvement of a charity in key steps of this process is still unusual. Illustrating the strategies and operational model… 

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License: This is an open access article under the terms of the Creative Commons Attribution 4

  • License: This is an open access article under the terms of the Creative Commons Attribution 4

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