Limitations and Challenges of Conducting Budget Impact Analyses in Rare Diseases: A Case Study of Alpha-1 Antitrypsin Deficiency

Editorial: Clinical Trial Design for Alpha-1 Antitrypsin Deficiency: A Model for Rare Diseases

Chronic Obstructive Pulmonary Diseases Journal of the COPD Foundation ◽

10.15326/jcopdf.2.2.2015.0140 ◽

2014 ◽

Vol 2 (2) ◽

pp. 91-93

Author(s):

Michael Jacobs ◽

Gerard Criner

Keyword(s):

Clinical Trial ◽

Rare Diseases ◽

Trial Design ◽

Clinical Trial Design ◽

Antitrypsin Deficiency ◽

Clinical Trial Design for Alpha-1 Antitrypsin Deficiency: A Model for Rare Diseases

Chronic Obstructive Pulmonary Diseases Journal of the COPD Foundation ◽

10.15326/jcopdf.2.2.2015.0132 ◽

2014 ◽

Vol 2 (2) ◽

pp. 177-190 ◽

Cited By ~ 2

Author(s):

Adam Wanner ◽

Stephen Groft ◽

J. Russell Teagarden ◽

Jeffrey Krischer ◽

Barry Davis ◽

...

Keyword(s):

Clinical Trial ◽

Rare Diseases ◽

Trial Design ◽

Clinical Trial Design ◽

Antitrypsin Deficiency ◽

The Importance of Reference Centers and Registries for Rare Diseases: The Example of Alpha-1 Antitrypsin Deficiency

COPD Journal of Chronic Obstructive Pulmonary Disease ◽

10.1080/15412555.2020.1795824 ◽

2020 ◽

Vol 17 (4) ◽

pp. 346-354

Author(s):

Marc Miravitlles ◽

Alexa Nuñez ◽

Maria Torres-Durán ◽

Francisco Casas-Maldonado ◽

Juan Luis Rodríguez-Hermosa ◽

...

Keyword(s):

Rare Diseases ◽

Antitrypsin Deficiency ◽

An electronic nose distinguishes the exhaled breath condensates of alpha-1-antitrypsin deficiency patients and healthy controls

Pneumologie ◽

10.1055/s-0029-1214113 ◽

2009 ◽

Vol 63 (S 01) ◽

Author(s):

AR Koczulla ◽

A Hattesohl ◽

S Noeske ◽

C Vogelmeier ◽

R Bals

Keyword(s):

Electronic Nose ◽

Exhaled Breath ◽

Healthy Controls ◽

Antitrypsin Deficiency ◽

Sweet's syndrome leading to acquired cutis laxa (Marshall's syndrome) in an infant with alpha 1-antitrypsin deficiency

Archives of Dermatology ◽

10.1001/archderm.131.10.1175 ◽

1995 ◽

Vol 131 (10) ◽

pp. 1175-1177 ◽

Cited By ~ 6

Author(s):

S. T. Hwang

Keyword(s):

Cutis Laxa ◽

Sweet’S Syndrome ◽

Sweet's Syndrome ◽

Antitrypsin Deficiency ◽

Liver Disease in Alpha-1 Antitrypsin Deficiency

Journal of Liver Research Disorders & Therapy ◽

10.15406/jlrdt.2015.01.00011 ◽

2015 ◽

Vol 1 (3) ◽

Author(s):

Craig Miller

Keyword(s):

Liver Disease ◽

Antitrypsin Deficiency ◽

Non-invasive assessment of hepatic fibrosis in a series of patients with alpha-1-antitrypsin deficiency

10.26226/morressier.57c5383dd462b80296c9bbf4 ◽

2016 ◽

Author(s):

Agnieszka Bakula

Keyword(s):

Hepatic Fibrosis ◽

Non Invasive ◽

Antitrypsin Deficiency ◽

Faculty Opinions recommendation of Portuguese consensus document for the management of alpha-1-antitrypsin deficiency.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.734531604.793576125 ◽

2020 ◽

Author(s):

Alice Turner

Keyword(s):

Consensus Document ◽

Antitrypsin Deficiency ◽

Faculty Opinions recommendation of The withdrawal of replacement therapy and outcomes in alpha-1 antitrypsin deficiency lung transplant recipients.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.735236487.793576141 ◽

2020 ◽

Author(s):

Alice Turner

Keyword(s):

Replacement Therapy ◽

Lung Transplant ◽

Transplant Recipients ◽

Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin ◽

Lung Transplant Recipients

Faculty Opinions recommendation of Activation of complement component 3 is associated with airways disease and pulmonary emphysema in alpha-1 antitrypsin deficiency.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.737245561.793576136 ◽

2020 ◽

Author(s):

Alice Turner

Keyword(s):

Pulmonary Emphysema ◽

Complement Component ◽

Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin ◽

Complement Component 3