Publications

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

Transthyretin Amyloidosis (ATTR)

Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis

Circulation

Author(s)

Scott Solomon, David Adams, Arnt Kristen, et al

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

Author(s)

Amy Simon, Farrah Pompilus, William Querbes, et al

Transthyretin Amyloidosis (ATTR)

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

New England Journal of Medicine

Author(s)

David Adams, Alejandra Gonzalez-Duarte, William O’Riordan, et al