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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The FDA recentlyapproved 2 drugs for adults with cardiomyopathy caused by transthyretin-mediated amyloidosis (ATTR), a rare and life-threatening disease. The oral medications, tafamidis meglumine and tafamidis, are the first to receive agency approval for this indication.
Source: JAMA - Category: General Medicine Source Type: research
Amyloidosis cutis dyschromica (ACD) is a clinicopathologic form of primary localized cutaneous amyloidosis (PLCA) which is considered to be autosomal recessive and characterized by prepubertal onset of reticular hyperpigmentation with hypopigmented spots, along with amyloid deposition in the papillary dermis. Recently, bi-allelic mutations in GPNMB, encoding glycoprotein (transmembrane) non-metastatic melanoma protein b have been described in ACD (Yang et al., 2018). In contrast, we describe three pedigrees with ACD in whom we identified semi-dominant GPNMB mutations thereby expanding the inheritance pattern of this disorder.
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Letters to the Editor Source Type: research
Source: Journal of Nuclear Cardiology - Category: Nuclear Medicine Source Type: research
AbstractCarpal tunnel syndrome (CTS) is a common finding among patients with cardiac amyloidosis. We sought to determine the prevalence of cardiac amyloidosis in patients who had undergone CTS surgery. From 2005 to 2014, 308 patients ≥ 60 years underwent CTS surgery. Of these, 233 (76%) agreed to participate in the study and 101 (73 ± 8 years; 68% females) showed left ventricular hypertrophy (LVH) ≥ 12 mm and underwent additional studies to diagnose AL and ATTR amyloidosis. Based on complementary studies, three p atients were diagnosed with cardiac amyloidosis (two wi...
Source: Journal of Cardiovascular Translational Research - Category: Cardiology Source Type: research
Source: Amyloid: The Journal of Protein Folding Disorders - Category: Biochemistry Authors: Source Type: research
Source: Amyloid: The Journal of Protein Folding Disorders - Category: Biochemistry Authors: Source Type: research
Nature Reviews Neurology, Published online: 17 June 2019; doi:10.1038/s41582-019-0210-4In this Review, Adams et al. discuss the latest insights into various aspects of hereditary transthyretin amyloidosis and its management, including its epidemiology, pathogenesis and the latest success with RNA interference and antisense oligonucleotide therapies.
Source: Nature Reviews Neurology - Category: Neurology Authors: Source Type: research
You're called by Ortho for a washout of infected knee hardware. They warn you the guy is a little sick. You review the record... 90 yo M with a PMHx of CHF (EF 25%), AFib, and 3rd degree AV block (all presumed due to amyloidosis) and COPD. He was admitted to an OSH a month ago for infected knee hardware and underwent arthroscopy x 3 and grew out pseudomonas. Went to SNF on cefepime, but redeveloped infected knee and got admitted to your hospital. He now has fluid overload with... Weekend Case
Source: Student Doctor Network - Category: Universities & Medical Training Authors: Tags: Anesthesiology Source Type: forums
Authors: Chen L, Luodelete M, Dong C, Li B, Zhang W, Nie P, Liu J, Chen X, Luo P Abstract Background: To investigate the pathological spectrum of glomerular disease in patients with renal insufficiency (RI) from 2008 to 2017. Methods and results: We calculated the estimated glomerular filtration rate (eGFR) with the Chronic Kidney Disease Epidemiology Collaboration creatinine (CKD-EPI) equation and defined RI as an eGFR
Source: Renal Failure - Category: Urology & Nephrology Tags: Ren Fail Source Type: research
Systemic amyloidosis is a devastating group of disorders for which there is no current cure. The treatment goal is to reduce the burden of amyloidogenic protein precursors. The treatment is only effective if a...
Source: Diagnostic Pathology - Category: Pathology Authors: Tags: Short report Source Type: research
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