The Rare Disease Moonshot: Paradigms Shift, Translational Medicine, and Regulatory Science for the World's Rarest Conditions.
Cécile OllivierSolange Corriol-RohouMarta Del ÁlamoRoseline FavresseJohanna KostenzerMathieu BoudesAnton E UssiKlaus VielR Michael LindenMagda ChlebusPublished in: Clinical pharmacology and therapeutics (2024)
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