A randomized, placebo-controlled study of givosiran in patients with acute hepatic porphyrias (ENVISION): Final (36-month) analysis of the Taiwan Cohort by Ming-Jen Lee, Hung-Chou Kuo, Lin-Na Chou, Marianne T. Sweetser, Jiaan-Der Wang

Subjects: Get full text

Kidney Involvement in Acute Hepatic Porphyrias: Pathophysiology and Diagnostic Implications by Andrea Ricci, Claudio Carmine Guida, Paola Manzini, Chiara Cuoghi, Paolo Ventura

Subjects: Get full text

A Comparison of Currently Approved Small Interfering RNA (siRNA) Medications to Alternative Treatments by Costs, Indications, and Medicaid Coverage by Inder Sehgal, Kevin Eells, Imani Hudson

Subjects: Get full text

Baseline urinary ALA and PBG as criteria for starting pharmacologic prophylactic treatment in acute intermittent porphyria treated with givosiran by Hung-Chou Kuo, Long-Sun Ro, Chia-Ni Lin, Chun-Che Chu, Ming-Feng Liao, Hong-Shiu Chang

Subjects: Get full text

Effectiveness and tolerability of givosiran for the management of acute hepatic porphyria: A monocenter real-life evaluation by Claudio Carmine Guida, Maria Nardella, Aurora del Mar YS Perez, Maria Savino, Gaetano Ferrara, Francesco Napolitano, Annalisa Crisetti, Francesco Aucella, Filippo Aucella

Subjects: Get full text

Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study by Bruce Wang, Paolo Ventura, Kei-ichiro Takase, Manish Thapar, David Cassiman, Ilja Kubisch, Shangbin Liu, Marianne T. Sweetser, Manisha Balwani

Subjects: Get full text

Brazilian registry of patients with porphyria: REBRAPPO study by Paulo Victor Sgobbi Souza, Gliciane Afonso, Wladimir Bocca Vieira de Rezende Pinto, Paulo de Lima Serrano, Bruno de Mattos Lombardi Badia, Igor Braga Farias, Ana Carolina dos Santos Jorge, Roberta Ismael Lacerda Machado, Icaro França Navarro Pinto, Glenda Barbosa Barros, Helvia Bertoldo de Oliveira, Samia Rogatis Calil, Cibele Franz, Acary Souza Bulle Oliveira

Subjects: Get full text

Idiosyncratic drug-induced liver injury caused by givosiran in a patient with acute intermittent porphyria by Christopher D. Ma, Denise Faust, Herbert L. Bonkovsky

Subjects: “…Givosiran…”
Get full text

Preventing hyperhomocysteinemia using vitamin B6 supplementation in Givosiran-treated acute intermittent porphyria: Highlights from a case report and brief literature review by Isabelle Redonnet-Vernhet, Patrick Mercié, Louis Lebreton, Jean-Marc Blouin, Didier Bronnimann, Samir Mesli, Claire Guibet, Emmanuel Ribeiro, Noémie Gensous, Pierre Duffau, Laurent Gouya, Emmanuel Richard

Subjects: Get full text

Spotlight on Givosiran as a Treatment Option for Adults with Acute Hepatic Porphyria: Design, Development, and Place in Therapy by Majeed CN, Ma CD, Xiao T, Rudnick S, Bonkovsky HL

Subjects: “…givosiran…”
Get full text

Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study by Eliane Sardh, Manisha Balwani, David C. Rees, Karl E. Anderson, Gang Jia, Marianne T. Sweetser, Bruce Wang

Subjects: Get full text

Elevated homocysteine is negatively correlated with plasma cystathionine β‐synthase activity in givosiran‐treated patients by Mark A. Keibler, Gautham V. Sridharan, Marianne T. Sweetser, Simina Ticau

Subjects: Get full text

Case Report: Lack of Response to Givosiran in a Case of ALAD Porphyria by Erica Graff, Karl E. Anderson, Cynthia Levy, Cynthia Levy

Subjects: Get full text

2019 FDA TIDES (Peptides and Oligonucleotides) Harvest by Danah Al Shaer, Othman Al Musaimi, Fernando Albericio, Beatriz G. de la Torre

Subjects: Get full text