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

Lucia Monaco, Lucia Faccio
2017 EMBO Molecular Medicine  
doi:10.15252/emmm.201607293 pmid:28148554 pmcid:PMC5331198 fatcat:b7hor75y5vdvlkrsibv57vofja