Alpha-1 antitrypsin (AAT) deficiency - what are the treatment options? (Q37614554)

21 August 2017

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title

Alpha-1 antitrypsin (AAT) deficiency - what are the treatment options? (English)

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alpha-1-antitrypsin deficiency

21 August 2017

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1

Ariel Modrykamien

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21 August 2017

James K Stoller

2

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PubMed ID

19827990

21 August 2017

language of work or name

English

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publication date

1 November 2009

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Expert Opinion on Pharmacotherapy

21 August 2017

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21 August 2017

10

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21 August 2017

16

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21 August 2017

2653-2661

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Alpha one antitrypsin deficiency: from gene to treatment

21 August 2017

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

A review of alpha-1 antitrypsin deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

What we owe to alpha(1)-antitrypsin and to Carl-Bertil Laurell

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Neonatal screening for alpha1-antitrypsin deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Alpha-1-antitrypsin deficiency. High prevalence in the St. Louis area determined by direct population screening

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Molecular confirmation of alpha 1-antitrypsin genotypes in newborn dried blood specimens

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Alpha1-antitrypsin deficiency. 2: genetic aspects of alpha(1)-antitrypsin deficiency: phenotypes and genetic modifiers of emphysema risk

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Alpha1-antitrypsin deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

A randomized trial comparing lung-volume-reduction surgery with medical therapy for severe emphysema

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Clinical features and natural history of severe alpha 1-antitrypsin deficiency. Roger S. Mitchell Lecture

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Biochemical, molecular characterization, and glycoproteomic analyses of alpha(1)-proteinase inhibitor products used for replacement therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Biochemical efficacy and safety of a new pooled human plasma alpha(1)-antitrypsin, Respitin.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Multi-center study: the biochemical efficacy, safety and tolerability of a new alpha1-proteinase inhibitor, Zemaira.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Does alpha1-antitrypsin augmentation therapy slow the annual decline in FEV1 in patients with severe hereditary alpha1-antitrypsin deficiency? Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL) alpha1-AT study group.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

A randomized clinical trial of alpha(1)-antitrypsin augmentation therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Exploring the role of CT densitometry: a randomised study of augmentation therapy in alpha1-antitrypsin deficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Augmentation therapy reduces frequency of lung infections in antitrypsin deficiency: a new hypothesis with supporting data

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

American Thoracic Society/European Respiratory Society statement: standards for the diagnosis and management of individuals with alpha-1 antitrypsin deficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Rate of progression of lung function impairment in alpha1-antitrypsin deficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Immune responses to recombinant adeno-associated virus vectors: putting preclinical findings into perspective

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Inhibiting polymerization: new therapeutic strategies for Z alpha1-antitrypsin-related emphysema.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Chemical chaperones mediate increased secretion of mutant alpha 1-antitrypsin (alpha 1-AT) Z: A potential pharmacological strategy for prevention of liver injury and emphysema in alpha 1-AT deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Polyethylene glycol conjugation at Cys232 prolongs the half-life of alpha1 proteinase inhibitor

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Fate of aerosolized recombinant DNA-produced alpha 1-antitrypsin: use of the epithelial surface of the lower respiratory tract to administer proteins of therapeutic importance

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Antibody response to aerosolized transgenic human alpha1-antitrypsin.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

Cost-effectiveness analysis of augmentation therapy for severe alpha1-antitrypsin deficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

alpha1-Antitrypsin augmentation therapy for PI*MZ heterozygotes: a cautionary note.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656560903300111

10.1517/14656560903300111

21 January 2018

Identifiers

DOI

1 reference

21 August 2017

1 reference

PubMed ID

19827990