Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues, Treatment of congenital alpha-1 antitrypsin deficiency

Case Medical Research ◽

10.31525/cmr-2a47e1d ◽

2020 ◽

Author(s):

Keyword(s):

Modified Nucleosides ◽

Orphan Designation ◽

Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin ◽

Sirna Oligonucleotide

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Orphan designation: Recombinant adeno-associated viral vector containing human alpha-1 antitrypsin gene, Treatment of congenital alpha-1 antitrypsin deficiency

Case Medical Research ◽

10.31525/cmr-29c9740 ◽

2020 ◽

Author(s):

Keyword(s):

Viral Vector ◽

Orphan Designation ◽

Gene Treatment ◽

Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin

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Orphan designation: Alpha-1 antitrypsin (inhalation use), Treatment of emphysema secondary to congenital alpha-1-antitrypsin deficiency

Case Medical Research ◽

10.31525/cmr-12043f0 ◽

2019 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin

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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 ◽

Alpha 1 Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin

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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 ◽

Alpha 1 Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin

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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 ◽

Alpha 1 Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin

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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 ◽

Alpha 1 Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin

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Orphan designation: synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues, Treatment of primary hyperoxaluria

Case Medical Research ◽

10.31525/cmr-de550b ◽

2019 ◽

Author(s):

Keyword(s):

Lactate Dehydrogenase ◽

Primary Hyperoxaluria ◽

Modified Nucleosides ◽

Orphan Designation ◽

Sirna Oligonucleotide

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Orphan designation: synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues, Treatment of primary hyperoxaluria

Case Medical Research ◽

10.31525/cmr-de7557 ◽

2019 ◽

Author(s):

Keyword(s):

Lactate Dehydrogenase ◽

Primary Hyperoxaluria ◽

Modified Nucleosides ◽

Orphan Designation ◽

Sirna Oligonucleotide

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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 ◽

Alpha 1 Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin

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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 Deficiency ◽

Alpha 1 Antitrypsin ◽

Lung Transplant Recipients

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