Cardiovascular (CV)
Renoprotective Effects of Small Interfering RNA Targeting Liver Angiotensinogen in Experimental Chronic Kidney Disease
Hypertension
Cardiovascular (CV)
No evidence for brain renin-angiotensin system activation during DOCA-salt hypertension
Clinical Science
Transthyretin Amyloidosis (ATTR)
Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
The Lancet Neurology
Acute Hepatic Porphyria (AHP)
Normal reference ranges for urinary δ-aminolevulinic acid and porphobilinogen levels
JIMD Reports
Transthyretin Amyloidosis (ATTR)
Transthyretin-stabilising mutation T119M is not associated with protection against vascular disease or death in the UK Biobank
Amyloid
Transthyretin Amyloidosis (ATTR)
Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates
Nucleic Acids Research
Acute Hepatic Porphyria (AHP)
Detecting rare diseases in electronic health records using machine learning and knowledge engineering: Case study of acute hepatic porphyria
PLOS One
Acute Hepatic Porphyria (AHP)
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria
New England Journal of Medicine
Transthyretin Amyloidosis (ATTR)
DISCOVERY: prevalence of transthyretin (TTR) mutations in a US-centric patient population suspected of having cardiac amyloidosis
Amyloid
Cardiovascular (CV)
RNA interference therapeutics targeting angiotensinogen ameliorate preeclamptic phenotype in rodent models
The Journal of Clinical Investigation
