Publications

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

Primary Hyperoxaluria Type 1 (PH1)

Characterising a healthy adult with a rare HAO1 knockout to support a therapeutic strategy for primary hyperoxaluria

eLife

Author(s)

Tracy L McGregor, Karen A Hunt, Elaine Yee, et al

Acute Hepatic Porphyria (AHP)

Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid, Givosiran, in Patients With Acute Hepatic Porphyria

Clinical Pharmacology and Therapeutics

Author(s)

Sagar Agarwal, Amy R Simon, Varun Goel, et al

Acute Hepatic Porphyria (AHP)

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

Hepatology

Author(s)

Laurent Gouya, Paolo Ventura, Manisha Balwani, et al

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

Author(s)

Xiaoping Zhang, Varun Goel, Husain Attarwala, et al

Cardiovascular (CV)

Strong and Sustained Antihypertensive Effect of Small Interfering RNA Targeting Liver Angiotensinogen

Hypertension

Author(s)

Estrellita Uijl, Katrina Mirabito Colafella, Yuan Sun, et al

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

Author(s)

Masatoshi Minamisawa, Brian Claggett, David Adams, et al

Acute Hepatic Porphyria (AHP)

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

New England Journal of Medicine

Author(s)

Eliane Sardh, Pauline Harper, Manisha Balwani, et al