Natural history and survival rate of familial amyloidosis with polyneuropathy: A nationwide databank
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DOI: 10.1002/acn3.51765
Abstract: Hereditary amyloid transthyretin (ATTRv) amyloidosis with polyneuropathy, a rare autosomal‐dominant disease, has gained attention in recent years owing to treatment improvements. However, epidemiological real‐world mega database of nationwide natural history and survival rates, especially with… read more here.
Keywords: natural history; amyloidosis polyneuropathy; history survival;
Chance or challenge, spoilt for choice? New recommendations on diagnostic and therapeutic considerations in hereditary transthyretin amyloidosis with polyneuropathy: the German/Austrian position and review of the literature
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DOI: 10.1007/s00415-020-09962-6
Abstract: Hereditary transthyretin amyloidosis is caused by pathogenic variants (ATTR v ) in the TTR gene. Alongside cardiac dysfunction, the disease typically manifests with a severely progressive sensorimotor and autonomic polyneuropathy. Three different drugs, tafamidis, patisiran,… read more here.
Keywords: recommendations diagnostic; transthyretin amyloidosis; diagnostic therapeutic; amyloidosis ... See more keywords
Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years
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DOI: 10.1007/s40120-020-00180-w
Abstract: Introduction The effects of tafamidis on mortality in Val30Met and non-Val30Met patients with transthyretin amyloidosis with polyneuropathy (ATTR-PN) were evaluated. Methods The analyses were based on cumulative data from the Val30Met patients in the 18-month… read more here.
Keywords: long term; transthyretin amyloidosis; treatment; mortality ... See more keywords
LONG-TERM, INTEGRATED CARDIAC SAFETY OF PATISIRAN IN PATIENTS WITH HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS WITH POLYNEUROPATHY
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DOI: 10.1016/s0735-1097(20)31317-6
Abstract: Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive, life-threatening disease. The majority of patients develop a mixed phenotype of polyneuropathy and cardiomyopathy. Patisiran is approved in certain countries globally for the treatment of hATTR amyloidosis with… read more here.
Keywords: transthyretin mediated; amyloidosis polyneuropathy; amyloidosis; hereditary transthyretin ... See more keywords
The impact of clinical heterogeneity on conducting network meta-analyses in transthyretin amyloidosis with polyneuropathy
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DOI: 10.1080/03007995.2020.1725742
Abstract: Abstract Objective: The comparative safety and efficacy of tafamidis, patisiran and inotersen treatments for transthyretin amyloidosis with polyneuropathy (ATTR-PN) has not been evaluated in clinical trials. In the absence of head-to-head evidence, indirect treatment comparisons… read more here.
Keywords: meta analyses; heterogeneity; transthyretin amyloidosis; clinical heterogeneity ... See more keywords
Hereditary Transthyretin Amyloidosis with Polyneuropathy: Monitoring and Management
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DOI: 10.2147/ijgm.s338430
Abstract: Abstract Our aim in this review is to discuss current treatments and investigational products and their effect on patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and provide suggestions for monitoring disease progression and treatment… read more here.
Keywords: monitoring management; hereditary transthyretin; amyloidosis polyneuropathy; transthyretin amyloidosis ... See more keywords