Therapies for rare diseases: therapeutic modalities, progress and challenges ahead
Erik Tambuyzer(Park Foundation), Benjamin Vandendriessche(Case Western Reserve University), Christopher P. Austin(National Institutes of Health), Philip J. Brooks(National Institutes of Health), Kristina Larsson(European Medicines Agency), Katherine I. Miller Needleman(United States Food and Drug Administration), James Valentine(Phelps Hospital), Kay E. Davies(University of Oxford), Stephen C. Groft(National Institutes of Health), Robert A. Preti(Hitachi (United States)), Tudor I. Oprea(University of New Mexico), Marco Prunotto(University of Geneva)
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