Cambridge's Akcea rebounds with FDA approval of first drug

After having one of its drugs rejected by the FDA in August, Cambridge biotech Akcea Therapeutics Inc. turned the tide on Friday, receiving approval for another treatment for a rare genetic disease. The FDA approved Tegsedi, known generically as inotersen, an RNA-based drug that is designed to treat a rare and often fatal genetic disease called hereditary ATTR amyloidosis, or hATTR amyloidosis. According to Akcea (Nasdaq: AKCA), the drug works by slowing down production of a protein that can build…
Source: Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news

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We report here, in simian immunodeficiency virus (SIV)+ rhesus macaques and patients diagnosed with HIV, brain region–specific up-regulation of amyloid precursor protein (APP) and Aβ (40 and 42) in astrocytes. In addition, we find increased expression of β-site cleaving enzyme (BACE1), APP, and Aβ in human primary astrocytes (HPAs) exposed to Tat. Mechanisms involved up-regulation of hypoxia- inducible factor (HIF-1α), its translocation and binding to the long noncoding RNA (lncRNA) BACE1‐antisense transcript (BACE1-AS), resulting, in turn, in the formation of the BACE1-AS/BACE1 RNA complex, su...
Source: PLoS Biology: Archived Table of Contents - Category: Biology Authors: Source Type: research
Source: Amyloid: The Journal of Protein Folding Disorders - Category: Biochemistry Authors: Source Type: research
ConclusionPatients newly diagnosed with ATTRv amyloidosis have substantial healthcare utilization and costs in the first year, primarily the initial months, post-diagnosis. Further research should examine later costs associated with disease progression and end-of-life care.
Source: Neurology and Therapy - Category: Neurology Source Type: research
Fibrillary glomerulonephritis (FGN) is distinguished from amyloidosis by thicker fibrils and the lack of staining with histochemical dyes typically reactive with amyloid. However, congophilic FGN has been prop...
Source: BMC Nephrology - Category: Urology & Nephrology Authors: Tags: Case report Source Type: research
Source: Journal of Nuclear Cardiology - Category: Nuclear Medicine Source Type: research
Blood Cancer Journal, Published online: 22 May 2020; doi:10.1038/s41408-020-0325-2The impact and importance of achieving a complete haematological response prior to renal transplantation in AL amyloidosis
Source: Blood Cancer Journal - Category: Hematology Authors: Source Type: research
Source: Amyloid: The Journal of Protein Folding Disorders - Category: Biochemistry Authors: Source Type: research
Abstract Amyloidosis comprises a group of disorders that accumulate modified autologous proteins in organs, mainly the kidneys. Few studies have addressed the amyloid compartmental distribution and associated clinical outcomes. The aim of this study was to present a case series of renal amyloidosis correlating histopathological data with glomerular filtration rate (GFR) during kidney biopsy. We studied 53 cases reviewed by nephropathologists from 2000 to 2018 in a single kidney biopsy center in Brazil. GFR was estimated using the CKD-EPI formula. Cases were divided into Group A ≥60 and Group B
Source: Braz J Med Biol Res - Category: Research Authors: Tags: Braz J Med Biol Res Source Type: research
We report the case of a Syrian patient who benefited from a partial hepatectomy for the treatment of multiple metastasis of a colorectal adenocarcinoma. Microscopic analysis of the surgical specimen revealed numerous amyloid deposits that did not match any of the usual forms of liver amyloidosis after immunohistochemistry typing. Some morphologic features of the deposits were highly suggestive of ALECT2. Complementary immunohistochemical study and mass spectrometry confirmed the diagnosis. PMID: 32446730 [PubMed - as supplied by publisher]
Source: Annales de Pathologie - Category: Pathology Authors: Tags: Ann Pathol Source Type: research
We report that CCL2 overexpression aggravated tau pathology in rTg4510 as shown by the increase in Gallyas stained neurofibrillary tangles as well as phosphorylated tau-positive inclusions. In addition, biochemical analysis showed a reduction in the levels of detergent-soluble tau species followed by increase in the insoluble fraction, indicating a shift toward larger tau aggregates. Indeed, increased levels of high molecular weight species of phosphorylated tau were found in the mice injected with CCL2. We also report that worsening of tau pathology following CCL2 overexpression was accompanied by a distinct inflammatory ...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
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