Advances in orphan drug development for alpha-1 antitrypsin deficiency: a 2025 update from the FDA and EMA
Therapeutic Advances in Respiratory Disease
Published online on January 07, 2026
Abstract
Therapeutic Advances in Respiratory Disease, Volume 20, January-December 2026.
Background:Research into safe and effective treatments for alpha-1 antitrypsin deficiency (AATD) has been ongoing for more than four decades. There is still a high medical need for better treatment options: Safe, effective, and convenient therapies that ...
Background:Research into safe and effective treatments for alpha-1 antitrypsin deficiency (AATD) has been ongoing for more than four decades. There is still a high medical need for better treatment options: Safe, effective, and convenient therapies that ...