Advances in orphan drug development for alpha-1 antitrypsin deficiency: a 2025 update from the FDA and EMA - Höger, Philipp; Ries, Markus; Olivares-Rivera, Arturo; Ersöz, Hilal; Buschulte, Katharina; Fähndrich, Sebastian; Kontogianni, Konstantina; Herth, Felix; Trudzinski, Franziska (Therapeutic advances in respiratory disease, 2026)
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