Patient-driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis.
Fondazione Telethon, Milan, Italy.
Lucia
0000-0001-5620-1790
L
Monaco
Fondazione Telethon, Milan, Italy.
Lucia
L
Faccio
D006801:Humans; D035583:Rare Diseases