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Journal of Proteomics|May 17, 2015
Vitamin D-binding protein as a biomarker of active disease in acute intermittent porphyriaIrantzu Serrano-Mendioroz, Ana Sampedro, María Isabel Mora, et al.Journal of Hepatology|May 24, 2016
Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyriaDelia D'Avola, Esperanza López-Franco, Bruno Sangro, et al.Liver Transplantation : Official Publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society|May 24, 2011
Liver transplantation for erythropoietic protoporphyria in EuropeStaffan Wahlin, Per Stal, Rene Adam, et al.Liver International : Official Journal of the International Association for the Study of the Liver|April 17, 2026
Clinical Characteristics and Outcomes of Acute Intermittent Porphyria: Insights From the European Porphyria RegistryCarl M Baravelli, Sverre Sandberg, Marte H Hammersland, et al.The New England Journal of Medicine|February 7, 2019
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent PorphyriaEliane Sardh, Pauline Harper, Manisha Balwani, et al.Journal of Internal Medicine|November 21, 2025
New cases of δ-aminolevulinic acid dehydratase deficiency: Functional insights into gene variants using an innovative mouse liver modelElena Di Pierro, Isabel Solares, Daniel Jericó, et al.Genetics in Medicine : Official Journal of the American College of Medical Genetics|May 11, 2019
International Porphyria Molecular Diagnostic Collaborative: an evidence-based database of verified pathogenic and benign variants for the porphyriasBrenden Chen, Sharon Whatley, Michael Badminton, et al.The Lancet. Haematology|May 2, 2026
Guidelines for the management of acute porphyria: recommendations from the International Porphyria NetworkYonatan Edel, Penelope E Stein, Hassan Kawtharany, et al.Hepatology (Baltimore, Md.)|September 13, 2019
EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent AttacksLaurent Gouya, Paolo Ventura, Manisha Balwani, et al.The New England Journal of Medicine|June 11, 2020
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent PorphyriaManisha Balwani, Eliane Sardh, Paolo Ventura, et al.Pageof 4