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

Dysregulation of homocysteine homeostasis in acute intermittent porphyria patients receiving heme arginate or givosiran

(2021) Jordi To‐Figueras et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Severe homocysteinemia in two givosiran-treated porphyria patients: is free heme deficiency the culprit?

(2021) Petro E. Petrides et al.   ANNALS OF HEMATOLOGY

Hyperhomocysteinemia in patients with acute porphyrias: a possible effect of ALAS1 modulation by siRNAm therapy and its control by vitamin supplementation

(2021) Andrea Ricci et al.   European Journal of Internal Medicine

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

(2021) Daphne Vassiliou et al.   JOURNAL OF INTERNAL MEDICINE

Hyperhomocysteinemia in patients with acute porphyrias: A potentially dangerous metabolic crossroad?

(2020) Paolo Ventura et al.   European Journal of Internal Medicine

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

(2020) Manisha Balwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

A phase 1/2 open label extension study of givosiran, an investigational RNAi therapeutic, in patients with acute intermittent porphyria

(2020) Penelope Stein et al.   JOURNAL OF HEPATOLOGY

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

(2019) Eliane Sardh et al.   NEW ENGLAND JOURNAL OF MEDICINE

Pathogenesis and clinical features of the acute hepatic porphyrias (AHPs)

(2019) Herbert L. Bonkovsky et al.   MOLECULAR GENETICS AND METABOLISM

Clinical Guide and Update on Porphyrias

(2019) Ulrich Stölzel et al.   GASTROENTEROLOGY

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

(2019) Laurent Gouya et al.   HEPATOLOGY

Recurrent attacks of acute hepatic porphyria: major role of the chronic inflammatory response in the liver

(2018) C. Schmitt et al.   JOURNAL OF INTERNAL MEDICINE

Acute hepatic porphyrias: Recommendations for evaluation and long-term management

(2017) Manisha Balwani et al.   HEPATOLOGY

Porphyria

(2017) D. Montgomery Bissell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Update review of the acute porphyrias

(2016) Penelope E. Stein et al.   BRITISH JOURNAL OF HAEMATOLOGY

Experiences and concerns of patients with recurrent attacks of acute hepatic porphyria: A qualitative study

(2016) Hetanshi Naik et al.   MOLECULAR GENETICS AND METABOLISM

High prevalence of and potential mechanisms for chronic kidney disease in patients with acute intermittent porphyria

(2015) Nicolas Pallet et al.   KIDNEY INTERNATIONAL

Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification

(2015) Amy Chan et al.   Molecular Therapy-Nucleic Acids

Acute Porphyrias in the USA: Features of 108 Subjects from Porphyrias Consortium

(2014) Herbert L. Bonkovsky et al.   AMERICAN JOURNAL OF MEDICINE

Management of acute intermittent porphyria

(2014) Pauline Harper et al.   Expert Opinion on Orphan Drugs

Liver Transplantation From Donors With Acute Intermittent Porphyria

(2013) Joanna K. Dowman et al.   ANNALS OF INTERNAL MEDICINE

The minimal clinically important difference in the Oxford knee score and Short Form 12 score after total knee arthroplasty

(2013) N. D. Clement et al.   KNEE SURGERY SPORTS TRAUMATOLOGY ARTHROSCOPY

The porphyrias: advances in diagnosis and treatment

(2012) M. Balwani et al.   BLOOD

Review of hepatocellular cancer, hypertension and renal impairment as late complications of acute porphyria and recommendations for patient follow-up

(2012) Mary Felicity Stewart   JOURNAL OF CLINICAL PATHOLOGY

The incidence of inherited porphyrias in Europe

(2012) George Elder et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Minimum clinically important difference in pain, disability, and quality of life after neural decompression and fusion for same-level recurrent lumbar stenosis: understanding clinical versus statistical significance

(2012) Scott L. Parker et al.   JOURNAL OF NEUROSURGERY-SPINE

Liver transplantation for acute intermittent porphyria is complicated by a high rate of hepatic artery thrombosis

(2011) Joanna K. Dowman et al.   LIVER TRANSPLANTATION

Porphyrias

(2010) Hervé Puy et al.   LANCET

Preliminary report: Hyperhomocysteinemia in patients with acute intermittent porphyria

(2010) Jordi To-Figueras et al.   METABOLISM-CLINICAL AND EXPERIMENTAL