Cargando…
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...
| Autores principales: | , |
|---|---|
| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley and Sons Inc.
2017
|
| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5331198/ https://www.ncbi.nlm.nih.gov/pubmed/28148554 http://dx.doi.org/10.15252/emmm.201607293 |