Augmentation therapy
- A survival study has just been completed, comparing matched AATD patients with lung disease in the U.S. with similar patients treated in the U.K.
- The only difference between the two groups was that U.S. patients received augmentation therapy for their lung disease, while the U.K. patients did not
- Matched on age, sex, lung function at diagnosis, genotype, and follow-up for at least 15 years, or until death or lung transplant
- Data sharing conformed to both U.S. and U.K. regulations
FDA/NIH public workshop: Developing Alpha-1 Antitrypsin Therapeutics – September 16, 2019
