2 documents found, page 1 of 1
Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense ...
Coelho, Teresa; Ando, Yukio; Benson, Merrill D.; Berk, John L.; Waddington-Cruz, Márcia; Dyck, Peter J.; Gillmore, Julian D.; Khella, Sami L.Introduction: AKCEA-TTR-LRx is a ligand-conjugated antisense (LICA) drug in development for the treatment of hereditary transthyretin amyloidosis (hATTR), a fatal disease caused by mutations in the transthyretin (TTR) gene. AKCEA-TTR-LRx shares the same nucleotide sequence as inotersen, an antisense medicine approved for use in hATTR polyneuropathy (hATTR-PN). Unlike inotersen, AKCEA-TTR-LRx is conjugated to a ...
In vitro and in vivo effects of SerpinA1 on the modulation of Transthyretin pro...
Bezerra, Filipa; Niemietz, Christoph; Schmidt, Hartmut H. J.; Zibert, Andree; Guo, Shuling; Monia, Brett P.; Gonçalves, Paula Isabel MeiraTransthyretin (TTR) proteolysis has been recognized as a complementary mechanism contributing to transthyretin-related amyloidosis (ATTR amyloidosis). Accordingly, amyloid deposits can be composed mainly of full-length TTR or contain a mixture of both cleaved and full-length TTR, particularly in the heart. The fragmentation pattern at Lys48 suggests the involvement of a serine protease, such as plasmin. The mos...