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Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

The New England journal of medicine, 2019-02, Vol.380 (6), p.549-558

2019

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Efficacy and safety of givosiran for acute hepatic porphyria: 24‐month interim analysis of the randomized phase 3 ENVISION study

Liver international, 2022-01, Vol.42 (1), p.161-172

2022

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Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study

Orphanet journal of rare diseases, 2024-10, Vol.19 (1), p.365-14, Article 365

2024

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S1170 Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyria: 24-Month Interim Analysis of the Phase 3 ENVISION Randomized Clinical Trial

The American journal of gastroenterology, 2021-10, Vol.116 (1), p.S545-S546

2021

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Unraveling mucolipidosis type III gamma through whole genome sequencing in late-onset retinitis pigmentosa: a case report

BMC ophthalmology, 2023-09, Vol.23 (1), p.1-394, Article 394

2023

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Challenges in the management of acute intermittent porphyria with recurrent attacks during pregnancy: A case report

CLINICAL CASE REPORTS, 2020-12, Vol.8 (12), p.2483-2487

2020

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RNAi therapy with givosiran significantly reduces attack rates in acute intermittent porphyria

Journal of internal medicine, 2022-05, Vol.291 (5), p.593-610

2022

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Homocysteine elevation in givosiran treatment: Suggested ALAS1 siRNA effect on cystathionine beta‐synthase

Journal of internal medicine, 2021-10, Vol.290 (4), p.928-930

2021

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Acute hepatic porphyria and maternal health: Clinical and biochemical follow‐up of 44 pregnancies

Journal of internal medicine, 2022-01, Vol.291 (1), p.81-94

2022

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Renal failure affects the enzymatic activities of the three first steps in hepatic heme biosynthesis in the acute intermittent porphyria mouse

PloS one, 2012-03, Vol.7 (3), p.e32978-e32978

2012

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Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

The New England journal of medicine, 2020-06, Vol.382 (24), p.2289-2301

2020

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Givosiran Likely Inhibits Cytochrome P450 More Substantially Than Reported

Clinical pharmacology and therapeutics, 2022-07, Vol.112 (1), p.24-24

2022

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Risk of primary liver cancer in acute hepatic porphyria patients: A matched cohort study of 1244 individuals

Journal of internal medicine, 2022-06, Vol.291 (6), p.824-836

2022

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Givosiran for Acute Intermittent Porphyria. Reply

The New England journal of medicine, 2020-11, Vol.383 (20), p.1989-1990

2020

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OS-122 - Final results from a phase 1/2, 48-month, open-label extension study of givosiran in patients with acute intermittent porphyria

Journal of hepatology, 2023-06, Vol.78, p.S92-S93

2023

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Porphyrin precursors and risk of primary liver cancer in acute intermittent porphyria: A case–control study of 188 patients

Journal of inherited metabolic disease, 2023-11, Vol.46 (6), p.1186-1194

2023

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Maternal and fetal outcomes in acute hepatic porphyria: A Swedish National Cohort S tudy

Journal of inherited metabolic disease, 2023-07, Vol.46 (4), p.675-686

2023

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Renal Injury Biomarkers Are Elevated in Acute Hepatic Porphyria: PO0467

Journal of the American Society of Nephrology, 2021-10, Vol.32 (10S), p.185-185

2021

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Comment on Ramai et al. Risk of Hepatocellular Carcinoma in Patients with Porphyria: A Systematic Review. ICancers/I 2022, I14/I, 2947

Cancers, 2023-01, Vol.15 (3)

2023

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Maternal and fetal outcomes in acute hepatic porphyria: A Swedish National Cohort Study

Journal of inherited metabolic disease, 2023-07, Vol.46 (4), p.675-686

2023

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Cancer risk in acute hepatic porphyria: a nationwide matched cohort study in 1, 245 individuals

JOURNAL OF HEPATOLOGY, 2022, Vol.77, p.S530

2022

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Final results from a phase 1/2, 48-month, open-label extension study of givosiran in patients with acute intermittent porphyria

JOURNAL OF HEPATOLOGY, 2023, Vol.78, p.S92-S93

2023

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A Drug‐Drug Interaction Study Evaluating the Effect of Givosiran, a Small Interfering Ribonucleic Acid, on Cytochrome P450 Activity in the Liver

Clinical pharmacology and therapeutics, 2021-11, Vol.110 (5), p.1250-1260

2021

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Risk for incident comorbidities, nonhepatic cancer and mortality in acute hepatic porphyria: A matched cohort study in 1244 individuals

Journal of inherited metabolic disease, 2023-03, Vol.46 (2), p.286-299

2023

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Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

Efficacy and safety of givosiran for acute hepatic porphyria: 24‐month interim analysis of the randomized phase 3 ENVISION study

Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study

S1170 Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyria: 24-Month Interim Analysis of the Phase 3 ENVISION Randomized Clinical Trial

Unraveling mucolipidosis type III gamma through whole genome sequencing in late-onset retinitis pigmentosa: a case report

Challenges in the management of acute intermittent porphyria with recurrent attacks during pregnancy: A case report

RNAi therapy with givosiran significantly reduces attack rates in acute intermittent porphyria

Homocysteine elevation in givosiran treatment: Suggested ALAS1 siRNA effect on cystathionine beta‐synthase

Acute hepatic porphyria and maternal health: Clinical and biochemical follow‐up of 44 pregnancies

Renal failure affects the enzymatic activities of the three first steps in hepatic heme biosynthesis in the acute intermittent porphyria mouse

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

Givosiran Likely Inhibits Cytochrome P450 More Substantially Than Reported

Risk of primary liver cancer in acute hepatic porphyria patients: A matched cohort study of 1244 individuals

Givosiran for Acute Intermittent Porphyria. Reply

OS-122 - Final results from a phase 1/2, 48-month, open-label extension study of givosiran in patients with acute intermittent porphyria

Porphyrin precursors and risk of primary liver cancer in acute intermittent porphyria: A case–control study of 188 patients

Maternal and fetal outcomes in acute hepatic porphyria: A Swedish National Cohort S tudy

Renal Injury Biomarkers Are Elevated in Acute Hepatic Porphyria: PO0467

Comment on Ramai et al. Risk of Hepatocellular Carcinoma in Patients with Porphyria: A Systematic Review. ICancers/I 2022, I14/I, 2947

Maternal and fetal outcomes in acute hepatic porphyria: A Swedish National Cohort Study

Cancer risk in acute hepatic porphyria: a nationwide matched cohort study in 1, 245 individuals

Final results from a phase 1/2, 48-month, open-label extension study of givosiran in patients with acute intermittent porphyria

A Drug‐Drug Interaction Study Evaluating the Effect of Givosiran, a Small Interfering Ribonucleic Acid, on Cytochrome P450 Activity in the Liver

Risk for incident comorbidities, nonhepatic cancer and mortality in acute hepatic porphyria: A matched cohort study in 1244 individuals

EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks

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