Rare diseases and effective treatments: are we delivering?

Lucio Luzzatto; Carla E. M. Hollak; Timothy M. Cox; Arrigo Schieppati; Christoph Licht; Helena Kääriäinen; Giampaolo Merlini; Franz Schaefer; Steven Simoens; Luca Pani; Silvio Garattini; Giuseppe Remuzzi

doi:10.1016/s0140-6736(15)60297-5

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