Impact of Patisiran, an RNAi Therapeutic, on Orthostatic Intolerance in Patients with Hereditary Transthyretin-Mediated Amyloidosis
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DOI: 10.1016/j.cardfail.2019.07.222
Abstract: Hereditary transthyretin-mediated (hATTR) amyloidosis is a rapidly progressive, multisystem disease which can impact numerous organs, including peripheral and autonomic nerves and heart. Orthostatic intolerance (OI), caused by amyloid deposition in the autonomic nervous system, is… read more here.
Keywords: impact; hereditary transthyretin; transthyretin mediated; hattr amyloidosis ... 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
Inotersen for the treatment of adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis
Sign Up to like & getrecommendations! Published in 2019 at "Expert Review of Clinical Pharmacology"
DOI: 10.1080/17512433.2019.1635008
Abstract: ABSTRACT Introduction: Hereditary transthyretin-mediated amyloidosis (ATTRv; v for variant) is an underdiagnosed, progressive, and fatal multisystemic disease with a heterogenous clinical phenotype that is caused by TTR gene mutations that destabilize the TTR protein, resulting… read more here.
Keywords: transthyretin mediated; pharmacology; hereditary transthyretin; mediated amyloidosis ... See more keywords
Liver‐directed drugs for transthyretin‐mediated amyloidosis
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DOI: 10.1111/jns.12519
Abstract: Transthyretin‐mediated amyloidosis (ATTR) is a rare, under‐recognized, progressively debilitating, fatal disease caused by the aggregation and extracellular deposition of amyloid transthyretin (TTR) fibrils in multiple organs and tissues throughout the body. TTR is predominantly synthesized… read more here.
Keywords: transthyretin mediated; ttr gene; mediated amyloidosis; gene ... See more keywords
The Silent Period for Small Fiber Sensory Neuropathy Assessment in a Mixed Cohort of Transthyretin-Mediated Amyloidosis
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DOI: 10.3390/biomedicines10092073
Abstract: Abstract Background: Transthyretin-mediated amyloidosis (ATTR) is a rare multisystemic disease involving the peripheral nervous system and heart. Autonomic and small fiber involvement is one of the hallmarks of ATTR, and many tools have been proposed… read more here.
Keywords: transthyretin mediated; small fiber; mediated amyloidosis; attr patients ... See more keywords