Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 17 publications
Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease.
Journal: The New England journal of medicine
Published: April 03, 2026
High resistance barrier and prophylactic protection in preclinical models of SARS-CoV-2 with two siRNA combination.
Journal: Nucleic acids research
Published: September 11, 2024
Patient Perspective on Acute Hepatic Porphyria with Sporadic Attacks: A Chronic Disease with Substantial Health-Related Quality of Life Impacts.
Journal: Advances in therapy
Published: March 11, 2022
A Drug-Drug Interaction Study Evaluating the Effect of Givosiran, a Small Interfering Ribonucleic Acid, on Cytochrome P450 Activity in the Liver.
Journal: Clinical pharmacology and therapeutics
Published: July 19, 2021
5-Aminolevulinate dehydratase porphyria: Update on hepatic 5-aminolevulinic acid synthase induction and long-term response to hemin.
Journal: Molecular genetics and metabolism
Published: August 31, 2020
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.
Journal: The New England journal of medicine
Published: June 11, 2020
Normal reference ranges for urinary δ-aminolevulinic acid and porphobilinogen levels.
Journal: JIMD reports
Published: February 22, 2020
Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid, Givosiran, in Patients With Acute Hepatic Porphyria.
Journal: Clinical pharmacology and therapeutics
Published: November 07, 2019
EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks.
Journal: Hepatology (Baltimore, Md.)
Published: May 10, 2019
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.
Journal: The New England journal of medicine
Published: February 07, 2019
Patient Perspective on Acute Intermittent Porphyria with Frequent Attacks: A Disease with Intermittent and Chronic Manifestations.
Journal: The patient
Published: June 20, 2018
A Highly Durable RNAi Therapeutic Inhibitor of PCSK9.
Journal: The New England journal of medicine
Published: May 04, 2017
Last Updated: 09/26/2026