Karri Penttila

Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective

S. Mariz, Angelo Loris Brunetta, A. Magrelli, 2021, British journal of clinical pharmacology.

Are products with an orphan designation for oncology indications different from products for other rare indications? A retrospective analysis of European orphan designations granted between 2002-2012

S. Simoens, I. Huys, M. Casteels, 2017, Orphanet Journal of Rare Diseases.