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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 (2015). Rare diseases and effective treatments: are we delivering?. , 385(9970), DOI: https://doi.org/10.1016/s0140-6736(15)60297-5.