PMID- 31550938
OWN - NLM
STAT- MEDLINE
DCOM- 20191024
LR  - 20191024
IS  - 1744-7658 (Electronic)
IS  - 1354-3784 (Linking)
VI  - 28
IP  - 10
DP  - 2019 Oct
TI  - Experimental and investigational drugs for the treatment of alpha-1 antitrypsin 
      deficiency.
PG  - 891-902
LID - 10.1080/13543784.2019.1672656 [doi]
AB  - Introduction: Alpha-1 antitrypsin deficiency (AATD) is most often associated with 
      chronic lung disease, early onset emphysema, and liver disease. The standard of 
      care in lung disease due to AATD is alpha-1 antitrypsin augmentation but there 
      are several new and emerging treatment options under investigation for both lung 
      and liver manifestations. Areas covered: We review therapeutic approaches to lung 
      and liver disease in alpha-1 antitrypsin deficiency (AATD) and the agents in 
      clinical development according to their mode of action. The focus is on products 
      in clinical trials, but data from pre-clinical studies are described where 
      relevant, particularly where progression to trials appears likely. Expert 
      opinion: Clinical trials directed at lung and liver disease separately are now 
      taking place. Multimodality treatment may be the future, but this could be 
      limited by treatment costs. The next 5-10 years may reveal new guidance on when 
      to use therapeutics for slowing disease progression with personalized treatment 
      regimes coming to the forefront.
FAU - Pye, Anita
AU  - Pye A
AD  - Institute of Applied Health Research, University of Birmingham , Birmingham , UK.
FAU - Turner, Alice M
AU  - Turner AM
AD  - Institute of Applied Health Research, University of Birmingham , Birmingham , UK.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20191011
PL  - England
TA  - Expert Opin Investig Drugs
JT  - Expert opinion on investigational drugs
JID - 9434197
RN  - 0 (Drugs, Investigational)
SB  - IM
MH  - Animals
MH  - Disease Progression
MH  - Drug Development/*methods
MH  - Drugs, Investigational/*administration & dosage/pharmacology
MH  - Humans
MH  - Liver Diseases/drug therapy/etiology
MH  - Lung Diseases/drug therapy/etiology
MH  - Precision Medicine/methods
MH  - alpha 1-Antitrypsin Deficiency/*drug therapy/physiopathology
OTO - NOTNLM
OT  - Alpha-1 antitrypsin deficiency
OT  - chronic obstructive pulmonary disease
OT  - cirrhosis
OT  - clinical trials
OT  - emphysema
EDAT- 2019/09/26 06:00
MHDA- 2019/10/28 06:00
CRDT- 2019/09/26 06:00
PHST- 2019/09/26 06:00 [pubmed]
PHST- 2019/10/28 06:00 [medline]
PHST- 2019/09/26 06:00 [entrez]
AID - 10.1080/13543784.2019.1672656 [doi]
PST - ppublish
SO  - Expert Opin Investig Drugs. 2019 Oct;28(10):891-902. doi: 
      10.1080/13543784.2019.1672656. Epub 2019 Oct 11.