Results 201 to 210 of about 21,545 (230)

Hereditäre Transthyretin-Amyloidosen

Der Nervenarzt, 2014
Hereditary amyloidosis is an autosomal dominant fatal multisystem disease caused by extracellular deposition of misfolded proteins and, therefore represents a hereditary protein folding or deposition disease that leads to progressive organ damage and eventually death.
openaire   +2 more sources

Liver transplantation and transthyretin amyloidosis

Muscle & Nerve, 2012
AbstractLiver transplantation as a specific treatment of transthyretin amyloidosis was first performed in 1990. The rationale for this treatment was that removal of the source (liver) of the amyloid precursor protein (mutated transthyretin) would stop progression of the disease. Indeed, after orthotopic liver transplantation (OLT), mutant transthyretin
openaire   +2 more sources

Novel Drugs Targeting Transthyretin Amyloidosis

Current Heart Failure Reports, 2014
Transthyretin amyloidosis (ATTR) is either a hereditary disease related to a mutation in the transthyretin gene that leads to neuropathy and/or cardiomyopathy or an acquired disease of the elderly that leads to restrictive cardiomyopathy. The prevalence of this disease is higher than once thought and awareness is likely to increase amongst physicians ...
openaire   +2 more sources

RNA-targeting and gene editing therapies for transthyretin amyloidosis

Nature Reviews Cardiology, 2022
Alberto Aimo, Vincenzo Castiglione, Maria Franzini   +2 more
exaly  

Transthyretin amyloidosis

Amyloid, 1996
Merrill D. Benson, Tomoyuki Uemichi
openaire   +1 more source

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

New England Journal of Medicine, 2021
Julian D Gillmore, Jӧrg Täubel, Marianna Fontana   +2 more
exaly  

Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis

New England Journal of Medicine, 2023
Mathew S Maurer, Robert L Gottlieb, Nobuhiro Tahara   +2 more
exaly  

Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

Lancet Neurology, The, 2021
Jonas Wixner, Arnt V Kristen, John L Berk   +2 more
exaly  

Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial

Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis, 2023
David Adams, Mark Taylor, Violaine Planté-Bordeneuve   +2 more
exaly  

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

New England Journal of Medicine, 2018
Chih-Chao Yang, Mitsuharu Ueda, Arnt V Kristen   +2 more
exaly