systemic amyloidoses

systemic amyloidoses

A group of diseases that have in common the deposition, outside the cells, of an abnormal, insoluble fibrillary protein called amyloid. In primary AMYLOIDOSIS the amyloid fibrils are monoclonal fragments of antibody (immunoglobulin) light chains. In secondary and familial amyloidoses other proteins, including apolipoprotein, fibrinogen, lysozyme and transthyretin, are involved.
References in periodicals archive ?
M2 PHARMA-July 31, 2018-Proclara Biosciences Initiates Clinical Trial of NPT189 for Systemic Amyloidoses
- US-based biotechnology company Proclara Biosciences has commenced a Phase 1a clinical trial of NPT189, a next-generation molecule is in development for the treatment of systemic amyloidoses, the company said.
The broad applicability of the GAIM technology enables the company to target multiple protein misfolding diseases, including neurodegenerative diseases and orphan systemic amyloidoses.
The systemic amyloidoses. N Engl J Med 1997;337(13):898-909.
(4,55,56,59-80) Typing the most common systemic amyloidoses (ie, AA, AL, AApol, Afib, ALys, and ATTR amyloid) could be done in the kidney biopsies.
In Europe, approximately 45% of systemic amyloidoses are AA amyloidosis.
However, recent advances in the treatment of systemic amyloidoses have changed this outlook and, hence, the importance of an early diagnosis of amyloid, and a correct diagnosis of its type, has been realized.
Although, at present, management of localized amyloid deposits is mainly conservative, treatment of systemic amyloidoses involves radical approaches ranging from aggressive chemotherapy to liver transplantation; new pharmacologic therapies targeting systemic fibrillogenesis are also in trials.
The broad applicability of the Proclara technology enables the company to target multiple protein misfolding diseases, including neurodegenerative diseases and several rare systemic amyloidoses. The lead GAIM drug candidate, NPT088, is in clinical development for the treatment of Alzheimer's disease.
Among systemic amyloidoses there are 3 sporadic forms and several familial forms.
Other hereditary systemic amyloidoses, including AApoAI, AGel, ALys, and AFib, are relatively rare.

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