Amyloidosis

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Related to ATTR amyloidosis: Hereditary amyloidosis, familial amyloidosis

amyloidosis

[‚am·ə·loi′dō·səs]
(medicine)
Deposition of amyloid in one or more organs of the body.

Amyloidosis

 

or amyloid dystrophy, a disorder of protein metabolism accompanied by the formation within tissues of a specific protein polysaccharide complex known as amyloid. The progress of the disease is associated with distortion of the protein-synthesizing function of the reticuloendothelial system and with accumulation of anomalous proteins in the blood plasma which act as autoantigens and also stimulate the formation of autoantibodies. As a result of antigen-antibody interaction, there is deposition of widely dispersed proteins which figure in the formation of amyloid. Once deposited in tissues, such as vascular and gland walls, the amyloid displaces functionally specialized elements of the organ; this process leads to the destruction of the organ.

Several types of amyloidosis are recognized: primary, secondary, and senile amyloidosis; amyloidosis with multiple myeloma; and localized tumorlike amyloidosis. Primary amyloidosis has no connection with any other disease; it affects mainly the cardiovascular system, alimentary tract, muscles, and skin. Secondary amyloidosis develops subsequent to other diseases which are accompanied by prolonged suppuration and tissue breakdown, such as tuberculosis, syphilis, and rheumatoid arthritis. It most often affects the spleen, liver, kidneys, adrenal glands, and intestines. Senile amyloidosis usually involves the heart. Amyloidosis may be either general or localized. Im-munodepressive and hepatic preparations are used in its treatment.

V. V. SEROV

References in periodicals archive ?
Akcea will have an on-site presence at the meeting at booth #207 where attendees can learn more about ATTR amyloidosis.
Systemic wild-type ATTR amyloidosis (formerly age-related or senile systemic amyloidosis) refers to the deposition of unmutated transthyretin in tissues, often in the myocardium, in elderly individuals.
PRX004, a potential treatment for ATTR amyloidosis, is a monoclonal antibody designed to target and clear the pathogenic, non-native forms of the TTR protein.
Based on Nobel Prize-winning science, ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made.
Patisiran, based on Nobel Prize-winning science, is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made.
With our planned APOLLO-B Phase 3 study to support potential expansion of ONPATTRO to ATTR amyloidosis patients with cardiomyopathy and advancement of subcutaneously administered vutrisiran in the HELIOS-A Phase 3 study, we are committing further efforts to help ATTR amyloidosis patients and to support our plans for the sustained and continued growth in our ATTR amyloidosis franchise for years to come," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam.
Alnylam will obtain global development and commercialization rights to its investigational RNAi therapeutics programs for the treatment of ATTR amyloidosis, including patisiran and ALN-TTRsc02.
ENPNewswire-June 19, 2019--Alnylam Announces Approval in Japan of ONPATTRO for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy
"We are encouraged that other therapeutics for ATTR amyloidosis have demonstrated benefit to patients by impacting the biological pathway leading to the formation of amyloid deposits, and we look forward to analyzing data from our PRX004 program which we expect will offer insights on a mechanism targeting the misfolded, pathogenic form of the TTR protein.
Patisiran is an investigational, intravenous RNAi therapeutic targeting transthyretin in development for the treatment of hereditary ATTR amyloidosis.