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Is augmentation therapy effective?

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