Results of a phase 1/2 study of cemdisiran in healthy subjects and patients with paroxysmal nocturnal hemoglobinuria.
Gaya A, Munir T, Urbano-Ispizua A, Griffin M, Taubel J, Bush J, Bhan I, Borodovsky A, Wang Y, Badri P, Garg P.
Gaya A, et al.
EJHaem. 2023 Jun 26;4(3):612-624. doi: 10.1002/jha2.748. eCollection 2023 Aug.
EJHaem. 2023.
PMID: 37601837
Free PMC article.
Cemdisiran, an RNA interference investigational treatment, silences complement component 5 (C5) expression in the liver. Previously reported results showed sustained reduction in C5 levels following cemdisiran monotherapy, with >90% reduction in patients with PNH …
Cemdisiran, an RNA interference investigational treatment, silences complement component 5 (C5) expression in the liver. Previously r …