Publications

Transthyretin Amyloidosis (ATTR)

Impact of Baseline Neuropathy Severity on Vutrisiran Treatment Response in the Phase 3 HELIOS-A Study

Neurology and Therapy

Author(s)

Marco Luigetti, Dianna Quan, John L. Berk, et al

Acute Hepatic Porphyria (AHP)

Elevated homocysteine is negatively correlated with plasma cystathionine β ‐synthase activity in givosiran‐treated patients

Journal of Inherited Metabolic Disease Reports

Author(s)

Mark A. Keibler, Gautham V. Sridharan, Marianne T. Sweetser, et al

Transthyretin Amyloidosis (ATTR)

Patisiran, an RNAi therapeutic for hereditary transthyretin-mediated amyloidosis: Sub-analysis in Taiwanese patients from the APOLLO study

Journal of the Formosan Medical Association

Author(s)

Lin KP, Yang CC, Lee YC, et al

Transthyretin Amyloidosis (ATTR)

RNA interference in the era of nucleic acid therapeutics

Nature Biotechnology

Author(s)

Vasant Jadhav, Akshay Vaishnaw, Kevin Fitzgerald, et al

Cardiovascular (CV)

RNA Interference With Zilebesiran for Mild to Moderate Hypertension: The KARDIA-1 Randomized Clinical Trial

The Journal of the American Medical Association

Author(s)

George L Bakris, Manish Saxena, Anil Gupta, et al

Transthyretin Amyloidosis (ATTR)

Impact of vutrisiran on exploratory cardiac parameters in hereditary transthyretin-mediated amyloidosis with polyneuropathy

European Journal of Heart Failure

Author(s)

Pablo Garcia-Pavia, Martha Grogan, Parag Kale, et al

Primary Hyperoxaluria Type 1 (PH1)

Distinguishing characteristics of pediatric patients with primary hyperoxaluria type 1 in PEDSnet

Journal of Pediatric Urology

Author(s)

Gregory E. Tasian, Kimberley Dickinson, Grace Park, et al

Primary Hyperoxaluria Type 1 (PH1)

Lumasiran: A Review in Primary Hyperoxaluria Type 1

Drugs

Author(s)

Connie Kang

Acute Hepatic Porphyria (AHP)

Clinical features of acute attacks, chronic symptoms, and long-term complications among patients with acute hepatic porphyria in Japan: a real-world claims database study

Orphanet Journal of Rare Diseases

Author(s)

Yutaka Horie, Yuka Yasuoka & Tomohide Adachi

Transthyretin Amyloidosis (ATTR)

Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis

New England Journal of Medicine

Author(s)

Mathew Maurer, Parag Kale, Marianna Fontana, et al