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. has turned the tide, 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 up…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news

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Condition:   Amyloidosis Intervention:   Procedure: Shear wave elastography Sponsors:   Fábio Fernandes;   Pfizer Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Authors: N M, Soni P, Anand R, Bali S, Hari Abstract Amyloidosis is a conglomeration of diseases due to production and deposition of amyloid, a proteinaceous substance, into organs, tissues, nerves and other places in the body affecting their normal function. This case report is of a 65 year old gentleman, resident of Bihar admitted with a short history of two months. He came with chief complaints of swelling in both lower limbs associated with heaviness in legs, shortness of breath, dizziness, fatigue and passage of frothy urine for two months. He was investigated and found to have proteinuria, low voltage ECG, Ec...
Source: Journal of the Association of Physicians of India - Category: General Medicine Tags: J Assoc Physicians India Source Type: research
Contributors : Richard M Giadone ; George J Murphy ; Taylor M MatteSeries Type : Expression profiling by high throughput sequencingOrganism : Homo sapiensThe systemic amyloidoses are diverse disorders in which misfolded proteins are secreted by effector organs and deposited as proteotoxic aggregates at downstream tissues. Although well-described clinically, the contribution of synthesizing organs to amyloid disease pathogenesis is unknown. Here, we utilize hereditary transthyretin amyloidosis (ATTR amyloidosis) induced pluripotent stem cells (iPSCs) to define the contribution of HLCs to the proteotoxicity of secreted TTR. ...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Expression profiling by high throughput sequencing Homo sapiens Source Type: research
Transthyretin and light-chain amyloidosis are the 2 main causes of cardiac amyloidosis. Recent developments in molecular imaging have transformed our ability to diagnose transthyretin cardiac amyloidosis noninvasively and unmasked a hitherto unrecognized prevalence of the disease. This review summarizes the current and evolving imaging approaches, their molecular structural basis, and the gaps in imaging capabilities that have arisen as a result of parallel developments in pharmacotherapy delivering the first effective treatment options for this condition.
Source: Journal of Nuclear Medicine - Category: Nuclear Medicine Authors: Tags: Focus on Molecular Imaging Source Type: research
Conclusion: 11C-metoclopramide can measure ABCB1 induction in the mouse brain without the need to consider an arterial input function and may find potential application in Alzheimer disease patients to noninvasively evaluate strategies to enhance the clearance properties of the BBB.
Source: Journal of Nuclear Medicine - Category: Nuclear Medicine Authors: Tags: Basic Source Type: research
Kalyani M Rapeti, Manoj Kamal, Bharat Paliwal, Deepak ModiIndian Journal of Anaesthesia 2020 64(7):640-642
Source: Indian Journal of Anaesthesia - Category: Anesthesiology Authors: Source Type: research
en IS Abstract OBJECTIVES: To investigate epidemiology, demography, and genetic and clinical characteristics of patients with familial Mediterranean fever (FMF) in Denmark. METHOD: In this population-based, cross-sectional cohort study, we identified FMF patients from discharge diagnoses using ICD-10 codes in the Danish National Patient Register, and linked data from the Danish Civil Registration System and laboratory databases for results of MEFV gene variant screening. RESULTS: We identified 495 FMF patients (prevalence 1:11 680) with a median age of 29 years and a female ratio of 51%. The me...
Source: Scandinavian Journal of Rheumatology - Category: Rheumatology Authors: Tags: Scand J Rheumatol Source Type: research
a Stoppacciaro Monoclonal Gammopathies of Renal Significance (MGRS) are a rather heterogeneous group of renal disorders caused by a circulating monoclonal (MC) immunoglobulin (Ig) component, often in the absence of multiple myeloma (MM) or another clinically relevant lymphoproliferative disorder. Nevertheless, substantial kidney damage could occur, despite the “benign” features of the bone-marrow biopsy. One example is renal amyloidosis, often linked to a small clone of plasma cells, without the invasive features of MM. However, patients with amyloidosis may present with a nephrotic syndrome and ren...
Source: Cancers - Category: Cancer & Oncology Authors: Tags: Review Source Type: research
Authors: Barge-Caballero G, Vázquez-García R, Barge-Caballero E, Couto-Mallón D, Paniagua-Martín MJ, Barriales-Villa R, Piñón-Esteban P, Bouzas-Mosquera A, Pombo-Otero J, Debén-Ariznavarreta G, Vázquez-Rodríguez JM, Crespo-Leiro MG Abstract INTRODUCTION AND OBJECTIVES: Light-chain amyloidosis (AL-CA) and transthyretin amyloidosis (ATTR-CA) are the most common types of cardiac amyloidosis (CA). We sought to study the clinical characteristics and prognosis of both diseases. METHODS: We conducted a single-centre, retrospective review of all patients di...
Source: Medicina Clinica - Category: General Medicine Tags: Med Clin (Barc) Source Type: research
Abstract Vutrisiran (ALN-TTRsc02) is a liver-directed, investigational, small interfering ribonucleic acid drug for the treatment of transthyretin (TTR)-mediated amyloidosis. This Phase 1, randomized, single-blind, placebo-controlled, single ascending dose study evaluated the pharmacodynamics, pharmacokinetics, and safety profile of subcutaneously-administered vutrisiran (5-300 mg) in healthy subjects (n = 80). Vutrisiran treatment achieved potent and sustained TTR reduction in a dose-dependent manner, with mean maximum TTR reduction of 57-97%, maintained for ≥90 days post-dose. Vutrisiran was rapidly absorbed ...
Source: Clinical Pharmacology and Therapeutics - Category: Drugs & Pharmacology Authors: Tags: Clin Pharmacol Ther Source Type: research
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