New insights into pathogenesis of acute porphyria attacks and proof-of-concept of augmenting hepatic PBGD as etiological treatment

by Jericó-Asenjo, D. (Daniel), Fontanellas-Romá, A. (Antonio)
Published 2022

Enzyme replacement therapy for acute intermittent porphyria and mRNA-based rescue in a new non-human primate model developed by long-term hepatic expression of iRNA

by Córdoba, K.M. (Karol M.), Fontanellas-Romá, A. (Antonio), Avila, M.A. (Matías Antonio)
Published 2023