Kamada reports positive top-line results of US Phase II trial of inhaled AAT therapy for AATD

Israeli-based therapeutics company Kamada has reported positive top-line results of its US Phase II clinical trial of inhaled Alpha-1 Antitrypsin (AAT) therapy to treat Alpha-1 Antitrypsin Deficiency (AATD).
Source: Drug Development Technology - Category: Pharmaceuticals Source Type: news

Related Links:

J Neurol Surg B Skull Base DOI: 10.1055/s-0040-1701237Aneurysms of the posterior circulation pose a unique challenge due to higher rupture rates, higher recurrence rates following endovascular treatment, and extended open cranial base approaches required to reach the ventrally located brainstem circulation. While endovascular therapy has made tremendous strides in successful treatment for most posterior circulation aneurysms, open microscopic approaches remain essential in specific circumstances. Here, we present a case of a patient who presented with acute, severe headache, and sixth nerve palsies, and who was found to ha...
Source: Journal of Neurological Surgery Part B: Skull Base - Category: Neurosurgery Authors: Tags: Skull Base: Operative Videos Source Type: research
McConkey S, Boland F, Galvin S, Kiernan F, O'Rourke J, Dwyer R, Power M, Geoghegan P, Larkin C, O'Leary RA, Freeman J, Gaffney A, Marsh B, Curley GF, McElvaney NG Abstract RATIONALE: Coronavirus disease 2019 (COVID-19) is a global threat to health. Its inflammatory characteristics are incompletely understood. OBJECTIVES: To define the cytokine profile of COVID-19, and to identify evidence of immunometabolic alterations in those with severe illness. METHODS: Levels of interleukin (IL)-1β, IL-6, IL-8, IL-10 and soluble TNF receptor 1 (sTNFR1) were assessed in plasma from healthy volunteers, hospitali...
Source: American Journal of Respiratory and Critical Care Medicine - Category: Respiratory Medicine Authors: Tags: Am J Respir Crit Care Med Source Type: research
AbstractPurpose of ReviewCommonly categorized as a rare disease, alpha-1 antitrypsin deficiency (AATD) is neither rare, when compared to many other genetic disorders, nor an actual disease, but rather a predisposition toward a wide variety of diseases. It is one of the most common genetic disorders which can lead to a spectrum of clinical manifestations, ranging from no symptoms to progressively debilitating systemic disease, most commonly affecting the lung and liver. It is therefore imperative for clinicians to recognize and be familiar with the spectrum of presentations, methods of diagnosis, and clinical management of ...
Source: Current Allergy and Asthma Reports - Category: Allergy & Immunology Source Type: research
Source: Gastroenterology - Category: Gastroenterology Authors: Source Type: research
Alfadda Thyroid hormones critically modulate body homeostasis and haemostasis by regulating energy and metabolism. Previous studies have focused on individual pathways or proteins that are affected by increases in thyroid hormone levels, while an overall plasma proteomic signature of this increased level is lacking. Herein, an integrated untargeted proteomic approach with network analysis was used to identify changes in circulating proteins in the plasma proteome between hyperthyroid and euthyroid states. Plasma from 10 age-matched subjects at baseline (hyperthyroid) and post treatment with carbimazole (euthyroid) was ...
Source: Molecules - Category: Chemistry Authors: Tags: Article Source Type: research
α1-Antitrypsin deficiency (AATD), characterised by reduced levels or functionality of α1-antitrypsin (AAT), is a significantly underdiagnosed genetic condition that predisposes individuals to lung and liver disease. Most of the available data on AATD are based on the most common, severe deficiency genotype (PI*ZZ); therefore, treatment and monitoring requirements for individuals with the PI*SZ genotype, which is associated with a less severe AATD, are not as clear. Recent genetic data suggest the PI*SZ genotype may be significantly more prevalent than currently thought, due in part to less frequent identificati...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Reviews Source Type: research
After the discovery of lung emphysema associated with α1-antitrypsin (AAT) deficiency (AATD) in 1963 by Laurell and Eriksson [1], basic and clinical advances in the AATD field have been dramatic and relatively fast [2]. It was soon confirmed that AATD was an inherited codominant recessive condition, but it took seven more years to associate AATD-related lung emphysema with neutrophil elastase [3, 4], and between 7 to 10 years to discover its causal relationship with childhood and adult liver cirrhosis [5, 6], and with neutrophilic panniculitis [7]. In the 1970s, isoelectric focusing (IEF) became the technique of...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Editorials Source Type: research
e S, Martinez-Delgado B Abstract Alpha-1 Antitrypsin deficiency (AATD) is an inherited condition characterized by reduced levels of serum Alpha-1 Antitrypsin (AAT) due to mutations in the SERPINA1 gene. The Pi*S (Glu264Val) is one of the most frequent deficient alleles of AATD, showing high incidence in the Iberian Peninsula. Herein, we describe two new alleles carrying S mutation, but producing null phenotype: QOVigo and QOAachen. The new alleles were identified by sequencing SERPINA1 gene in three patients who had lower AAT serum levels than expected for the initial genotype. These alleles are the result of comb...
Source: Am J Respir Cell Mol... - Category: Respiratory Medicine Authors: Tags: Am J Respir Cell Mol Biol Source Type: research
Authors: Rodrigues JF, Mineiro A, Reis A, Ventura DG, Fernandez-Llimos F, Costa F, Gomes J, Silva JM, Lopes P, Cordeiro CR Abstract Alpha-1 antitrypsin deficiency is an autosomal co-dominant inherited disorder that results in decreased circulating levels of alpha-1 antitrypsin (also known as alpha-1 proteinase inhibitor) and predisposes affected individuals to early onset lung and liver disease. There is currently no cure for alpha-1 antitrypsin deficiency. However, appropriate treatment and a high standard of clinical care can prevent patients from being seriously affected and having to undergo majo...
Source: Acta Medica Portuguesa - Category: General Medicine Tags: Acta Med Port Source Type: research
Authors: Greulich T, Fähndrich S, Clarenbach C, Gleiber W, Hautmann H, Heine R, Idzko M, Schmidt-Scherzer K, Skowasch D, Wiewrodt R, Bals R, Koczulla AR PMID: 32492720 [PubMed - as supplied by publisher]
Source: Pneumologie - Category: Respiratory Medicine Tags: Pneumologie Source Type: research
More News: Alpha-1 Antitrypsin Deficiency | Clinical Trials | Pharmaceuticals