FDA OKs Inotersen for Hereditary ATTR With Polyneuropathy FDA OKs Inotersen for Hereditary ATTR With Polyneuropathy

Inotersen treatment led to improvements in neurologic and quality-of-life endpoints in patients with hereditary transthyretin amyloidosis with polyneuropathy in a pivotal phase 3 trial.FDA Approvals
Source: Medscape Medical News Headlines - Category: Consumer Health News Tags: Rheumatology News Alert Source Type: news

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Abstract Light chain (AL) amyloidosis is the most common type of systemic amyloidosis, affecting around 10 people per million per year. In Europe, approximately 5000 new diagnosis per year are reported. Deposition of amyloid fibrils derived from antibody light chains are key pathogenic agents in AL amyloidosis. They can be deposited in multiple organs but cardiac involvement carries a major risk of mortality. The prognosis is poor in cases associated with multiple myeloma. The average survival is around 1 year. Up to half of all patients with cardiac amyloidosis die suddenly; 75% ofthose deaths are due to heart fa...
Source: International Journal of Clinical and Experimental Pathology - Category: Pathology Authors: Tags: Int J Clin Exp Pathol Source Type: research
Condition:   AL Amyloidosis Interventions:   Drug: CAEL-101;   Other: Placebo;   Drug: cyclophosphamide, bortezomib, and dexamethasone (CyBorD) Sponsors:   Caelum Biosciences;   IQVIA Biotech Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Publication date: Available online 7 August 2020Source: Revista Española de Cardiología (English Edition)Author(s): Tomás Ripoll-Vera, Jorge Álvarez Rubio, Mercedes Iglesias, Inés Losada López, Asunción Ferrer-Nadal, Juan González Moreno
Source: Revista Espanola de Cardiologia - Category: Cardiology Source Type: research
We present a case of 50-year-old man with lymphadenopathy, which was later confirmed to be amyloidosis on biopsy and serum-free light chain assay with efficacious use of 18F-FDG PET/CT for response assessment to bortezomib, cyclophosphamide, and dexamethasone.
Source: Clinical Nuclear Medicine - Category: Nuclear Medicine Tags: Interesting Images Source Type: research
Authors: Sekiguchi Y, Wakabayashi M, Iizuka H, Takizawa H, Sugimoto K, Sakajiri S, Inano T, Fukuda Y, Hamano Y, Tomita S, Izumi H, Isogai H, Okubo M, Nakamura N, Sawada T, Matsumoto K, Noguchi M Abstract A 47-year-old male with macroglossia presented with dyspnea on effort and chest pain at rest. Cardiac MRI revealed diffuse global subendocardial late gadolinium enhancement below the left ventricular endocardium and a dark blood pool of intracardiac contrast medium. Tongue biopsy revealed amyloid deposition, which was limited in the myocardium. He was diagnosed with primary light chain amyloidosis. His condition wa...
Source: Journal of Clinical and Experimental Hematopathology : JCEH - Category: Hematology Tags: J Clin Exp Hematop Source Type: research
AbstractHereditary variant transthyretin amyloidosis (ATTRv) is a rare genetic defect that affects about 5000 –10,000 people worldwide, causing amyloidosis secondary to misfolding of mutant transthyretin (TTR) protein fibrils. TTR mutations can cause protein deposits in many extracellular regions of organs, but those deposits in cardiac and axonal cells are the primary cause of this clinical syndrome. Tre atment options are limited, but new drugs are being developed. Patisiran, a novel drug, is a liposomal siRNA against TTR that specifically targets this protein, reducing the accumulation of TTR in tissues, with subs...
Source: Neurology and Therapy - Category: Neurology Source Type: research
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Source: Amyloid: The Journal of Protein Folding Disorders - Category: Biochemistry Authors: Source Type: research
AbstractPurpose of ReviewTo review the uses of echocardiography in patients with cancer and how it has expanded beyond the typical monitoring of systolic function during potentially cardiotoxic cancer therapeutics.Recent FindingsIn addition to myocardial strain imaging being a predictor of subsequent left ventricular dysfunction, it can be used for pattern recognition to help identify patients with cardiac amyloidosis or Takotsubo cardiomyopathy. Echocardiography is essential for diagnosis and planning of intervention for aortic stenosis in radiation-induced valvular disease, for which transcutaneous aortic valve replaceme...
Source: Current Cardiology Reports - Category: Cardiology Source Type: research
AbstractPurpose of ReviewTo provide a functional review for practicing clinicians on the current and emerging treatment considerations for transthyretin (TTR) cardiac amyloidosis (ATTR-CA).Recent FindingsCurrent treatment considerations are characterized as those silencing TTR translation, stabilizing TTR tetramers, and disrupting amyloid fibril deposition.SummaryHistorically considered a rare disease state, ATTR-CA is increasingly recognized as an important mediator of heart failure morbidity and mortality. The emergence of widely available therapies for ATTR-CA has developed hope for patients where little was previously ...
Source: Current Cardiology Reports - Category: Cardiology Source Type: research
Condition:   AL Amyloidosis Interventions:   Drug: CAEL-101;   Other: Placebo;   Drug: cyclophosphamide, bortezomib, and Dexamethasone (CyBorD) Sponsors:   Caelum Biosciences;   IQVIA Biotech Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
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