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Patient‐driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis
The recent approval of Strimvelis, the first ex vivo gene therapy to gain marketing authorization (Schimmer & Breazzano, 2016), 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...
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| Publicado en: | EMBO Mol Med |
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| Autores principales: | , |
| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
John Wiley and Sons Inc.
2017
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| Materias: | |
| Acceso en línea: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5331198/ https://ncbi.nlm.nih.gov/pubmed/28148554 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201607293 |
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