Transthyretin Amyloidosis (ATTR)
Impact of Baseline Neuropathy Severity on Vutrisiran Treatment Response in the Phase 3 HELIOS-A Study
Neurology and Therapy
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
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
Transthyretin Amyloidosis (ATTR)
RNA interference in the era of nucleic acid therapeutics
Nature Biotechnology
Cardiovascular (CV)
RNA Interference With Zilebesiran for Mild to Moderate Hypertension: The KARDIA-1 Randomized Clinical Trial
The Journal of the American Medical Association
Transthyretin Amyloidosis (ATTR)
Impact of vutrisiran on exploratory cardiac parameters in hereditary transthyretin-mediated amyloidosis with polyneuropathy
European Journal of Heart Failure
Primary Hyperoxaluria Type 1 (PH1)
Distinguishing characteristics of pediatric patients with primary hyperoxaluria type 1 in PEDSnet
Journal of Pediatric Urology
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
Transthyretin Amyloidosis (ATTR)
Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis
New England Journal of Medicine
