Rare diseases and effective treatments: are we delivering?

  title={Rare diseases and effective treatments: are we delivering?},
  author={Lucio Luzzatto and Carla E. M. Hollak and Timothy Martin Cox and Arrigo Schieppati and Christoph Licht and Helena K{\"a}{\"a}ri{\"a}inen and Giampaolo Merlini and Franz S. Schaefer and Steven Simoens and L. Pani and Silvio Garattini and Giuseppe Remuzzi},
  volume={385 9970},
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