Results 151 to 160 of about 141,957 (168)

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

New England Journal of Medicine, 2018
BACKGROUND Hereditary transthyretin amyloidosis is caused by pathogenic single-nucleotide variants in the gene encoding transthyretin (TTR) that induce transthyretin misfolding and systemic deposition of amyloid.
Laura Obici, Giampaolo Merlini, Josep Gámez   +2 more
exaly   +2 more sources

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Neurological manifestations of hereditary transthyretin amyloidosis: a focus on diagnostic delays

Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis, 2022
John L Berk, Lawreen Connors, Vaishali Sanchorawala   +2 more
exaly  

CSF/plasma levels, transthyretin stabilisation and safety of multiple doses of tolcapone in subjects with hereditary ATTR amyloidosis

Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis, 2022
Masahide Yazaki, Yoshiki Sekijima, Ken Takasone   +2 more
exaly  

Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis

Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis, 2020
Julian D Gillmore, Laura Obici, Arnt V Kristen   +2 more
exaly  

A simple core dataset and disease severity score for hereditary transthyretin (ATTRv) amyloidosis

Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis, 2021
Julian D Gillmore, Alexander Hardy, Jonas Wixner   +2 more
exaly  

Indirect treatment comparison of the efficacy of patisiran and inotersen for hereditary transthyretin-mediated amyloidosis with polyneuropathy

Expert Opinion on Pharmacotherapy, 2021
Gautam Sajeev, Peter Gorevic, Jaclyn Franklin   +2 more
exaly  

Real-world outcomes in non-endemic hereditary transthyretin amyloidosis with polyneuropathy: a 20-year German single-referral centre experience

Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis, 2021
Arnt V Kristen, Christoph Kimmich, Stefan Schönland   +2 more
exaly  

Quantification of cardiac amyloid with [18F]Flutemetamol in patients with V30M hereditary transthyretin amyloidosis

Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis, 2020
Ole B. Suhr, Jan Axelsson, Björn Pilebro   +2 more
exaly  

Societal costs and burden of hereditary transthyretin amyloidosis polyneuropathy

Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis, 2020
Mamede de Carvalho, João Costa, Isabel Conceição   +2 more
exaly  

Assessment of patients with hereditary transthyretin amyloidosis – understanding the impact of management and disease progression

Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis, 2019
Laura Obici, Yesim Parman, Lucía Galán Dávila   +2 more
exaly