The total number of rare conditions is debated, partly because of the variety of definitions of what constitutes rare. A broader consensus view of what rare means, based on improved understanding of individual group and patient clinicopathological characteristics, will help maximize the impact of technological advances in therapeutic development programmes.
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Therapies Sci Comm, Int Rare Dis Res Consortium, Paris, France
Assoc British Pharmaceut Ind, London, EnglandTherapies Sci Comm, Int Rare Dis Res Consortium, Paris, France
O'Connor, Daniel J.
Gabaldo, Michela
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Therapies Sci Comm, Int Rare Dis Res Consortium, Paris, France
Aptuit Evotec Co, Verona, ItalyTherapies Sci Comm, Int Rare Dis Res Consortium, Paris, France
Gabaldo, Michela
Aartsma-Rus, Annemieke
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Therapies Sci Comm, Int Rare Dis Res Consortium, Paris, France
Leiden Univ, Med Ctr, Dept Human Genet, Leiden, Netherlands
Dutch Ctr RNA Therapeut, Leiden, NetherlandsTherapies Sci Comm, Int Rare Dis Res Consortium, Paris, France
Aartsma-Rus, Annemieke
Jonker, Anneliene Hechtelt
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Therapies Sci Comm, Int Rare Dis Res Consortium, Paris, France
Univ Twente, Enschede, NetherlandsTherapies Sci Comm, Int Rare Dis Res Consortium, Paris, France