Publications

Cardiovascular (CV)

Renoprotective Effects of Small Interfering RNA Targeting Liver Angiotensinogen in Experimental Chronic Kidney Disease

Hypertension

Author(s)

Dominique Bovée, Liwei Ren, Estrellita Uijl, et al

Cardiovascular (CV)

No evidence for brain renin-angiotensin system activation during DOCA-salt hypertension

Clinical Science

Author(s)

Estrellita Uijl, Liwei Ren, Katrina Mirabito Colafella, et al

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

Author(s)

David Adams, Michael Polydefkis, Alejandra González-Duarte, et al

Acute Hepatic Porphyria (AHP)

Normal reference ranges for urinary δ-aminolevulinic acid and porphobilinogen levels

JIMD Reports

Author(s)

Sagar Agarwal, Bahru Habtemarium, Yuanxin Xu, et al

Transthyretin Amyloidosis (ATTR)

Transthyretin-stabilising mutation T119M is not associated with protection against vascular disease or death in the UK Biobank

Amyloid

Author(s)

Margaret M Parker, Simina Ticau, James Butler, et al

Transthyretin Amyloidosis (ATTR)

Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates

Nucleic Acids Research

Author(s)

Christopher Brown, Swati Gupta, June Qin, et al

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

Author(s)

Aaron M Cohen, Steven Chamberlin, Thomas Deloughery, et al

Acute Hepatic Porphyria (AHP)

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

New England Journal of Medicine

Author(s)

Manisha Balwani, Eliane Sardh, Paolo Ventura, et al

Transthyretin Amyloidosis (ATTR)

DISCOVERY: prevalence of transthyretin (TTR) mutations in a US-centric patient population suspected of having cardiac amyloidosis

Amyloid

Author(s)

Ola Akinboboye, Keyur Shah, Alberta L. Warner, et al.

Cardiovascular (CV)

RNA interference therapeutics targeting angiotensinogen ameliorate preeclamptic phenotype in rodent models

The Journal of Clinical Investigation

Author(s)

Nadine Haase, Donald Foster, Mark Cunningham, et al