Primary Hyperoxaluria Type 1 (PH1)
Characterising a healthy adult with a rare HAO1 knockout to support a therapeutic strategy for primary hyperoxaluria
eLife
Acute Hepatic Porphyria (AHP)
Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid, Givosiran, in Patients With Acute Hepatic Porphyria
Clinical Pharmacology and Therapeutics
Acute Hepatic Porphyria (AHP)
EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks
Hepatology
Transthyretin Amyloidosis (ATTR)
Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure-Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis
Journal of Clinical Pharmacology
Cardiovascular (CV)
Strong and Sustained Antihypertensive Effect of Small Interfering RNA Targeting Liver Angiotensinogen
Hypertension
Transthyretin Amyloidosis (ATTR)
Association of Patisiran, an RNA Interference Therapeutic, With Regional Left Ventricular Myocardial Strain in Hereditary Transthyretin Amyloidosis: The APOLLO Study
JAMA Cardiology
Acute Hepatic Porphyria (AHP)
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
New England Journal of Medicine
Transthyretin Amyloidosis (ATTR)
Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis
Circulation
Acute Hepatic Porphyria (AHP)
Patient Perspective on Acute Intermittent Porphyria with Frequent Attacks: A Disease with Intermittent and Chronic Manifestations
The Patient: Patient-Centered Outcomes Research
Transthyretin Amyloidosis (ATTR)
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
New England Journal of Medicine
