This work has been partially supported by ANR-funded I-BioLex project (ANR-20-CE26-0007-01) and by H2020-funded EuroGCT project (GA965241). 1 Orphan medicinal products are intended to treat diseases which are rare. Because they are rare, these diseases have tended to be neglected by the pharmaceutical industry in favour of more financially attractive medicines markets. Even if patients suffering from orphan diseases are, by definition, rare, they should be benefitting from quality treatments as any other patient in the European Union (EU). 2 Yet, orphan disease...
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