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: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news

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Amyloid deposition of WT human β2-microglobulin (WT-hβ2m) in the joints of long-term hemodialysis patients is the hallmark of dialysis-related amyloidosis. In vitro, WT-hβ2m does not form amyloid fibrils at physiological pH and temperature unless co-solvents or other reagents are added. Therefore, understanding how fibril formation is initiated and maintained in the joint space is important for elucidating WT-hβ2m aggregation and dialysis-related amyloidosis onset. Here, we investigated the roles of collagen I and the commonly administered anticoagulant, low-molecular-weight (LMW) heparin, in the initia...
Source: Journal of Biological Chemistry - Category: Chemistry Authors: Tags: Molecular Bases of Disease Source Type: research
Publication date: Available online 12 June 2019Source: Urology Case ReportsAuthor(s): Puja Patel, Mohammad Pourmandi, Jeffrey S. LombardAbstractPrimary amyloidosis of the bladder is a rare clinical occurrence, with only a limited number of cases documented in literature thus far. Herein, is the presentation of a case involving a 75 year old male who presented with painless gross hematuria. Subsequent laboratory results and imaging completed were misleading, creating a high suspicion for bladder malignancy. Further workup revealed bladder biopsies to have positive apple-green birefringence with Congo-red immunostaining, dia...
Source: Urology Case Reports - Category: Urology & Nephrology Source Type: research
Conditions:   Valve Stenoses, Aortic;   Amyloidosis Cardiac Intervention:   Diagnostic Test: Diagnosis of amyloidosis Sponsor:   Javier López Pais Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Condition:   Amyloidosis Intervention:   Diagnostic Test: Amyloidosis typing using mass spectrometry-based proteomics Sponsors:   University Hospital, Toulouse;   Proteomics and Mass Spectrometry of Biomolecules, Proteomics Infrastructure of Toulouse Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Conditions:   Valve Stenoses, Aortic;   Amyloidosis Cardiac Intervention:   Diagnostic Test: Diagnosis of amyloidosis Sponsor:   Javier López Pais Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Condition:   Amyloidosis Intervention:   Diagnostic Test: Amyloidosis typing using mass spectrometry-based proteomics Sponsors:   University Hospital, Toulouse;   Proteomics and Mass Spectrometry of Biomolecules, Proteomics Infrastructure of Toulouse Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Insulin-derived amyloidosis is a skin-related complication of insulin therapy that interferes with insulin therapy. Although toxicities of in vitro-formed insulin amyloid fibrils have been well studied, the to...
Source: BMC Endocrine Disorders - Category: Endocrinology Authors: Tags: Case report Source Type: research
We examined 49,693 case reports from the Japanese Society of Internal Medicine database. A total of 2,893 reports were included after excluding 46,022 reports that did not include the term "heart failure" and 778 reports with no indications of symptoms of heart failure. We assessed each patient's basal disease, and according to the abstracts, we reported their prognosis as dead or alive. Results Of the 2,893 reports included, 1,952 (67.5%) and 941 (32.5%) had cardiovascular and non-cardiovascular diseases as the causes, respectively; these cases were attributed to 725 different diseases, 196 (27.0%) and 529 (73.0...
Source: Internal Medicine - Category: Internal Medicine Tags: Intern Med Source Type: research
We report the clinical features of a patient with hereditary ATTR amyloidosis associated with a novel mutation (Y114S, p.Y134S). A 65-year-old Japanese man was admitted to our hospital after a 3-year history of progressive dyspnea on exertion. Five years previously, he presented dysesthesia in both hands caused by carpal tunnel syndrome. A genetic analysis revealed a base pair substitution of adenine to cytosine in the second codon of exon 4, residue 114, in the TTR gene (c.401A>C). The clinical characteristics were progressive cardiomyopathy with a poor vital prognosis, late onset, sporadic case, bilateral carpal tunne...
Source: Internal Medicine - Category: Internal Medicine Tags: Intern Med Source Type: research
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Source: Amyloid: The Journal of Protein Folding Disorders - Category: Biochemistry Authors: Source Type: research
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