The Rare Disease Moonshot: Paradigms Shift, Translational Medicine, and Regulatory Science for the World’s Rarest Conditions : Clinical Pharmacology and Therapeutics

Cécile Ollivier 1, * , Solange Corriol- Rohou 2 , Marta del Álamo3, Roseline Favresse 4, Johanna Kostenzer 5 , Mathieu Boudes6 et....al.2024-09-032024-09-032024-09-03http://98.70.32.231:4000/handle/123456789/1340The Rare Disease Moonshot: Paradigms Shift, Translational Medicine, and Regulatory Science for the World’s Rarest Conditions : Clinical Pharmacology and TherapeuticsArticle