Primary Hyperoxaluria Type 1 (PH1)
Reducing diagnostic delays in acute hepatic porphyria using health records data and machine learning
Journal of the American Medical Informatics Association
Transthyretin Amyloidosis (ATTR)
Effectiveness of patisiran after switching from tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy
European Journal of Neurology
Transthyretin Amyloidosis (ATTR)
Six-minute walk test as clinical end point in cardiomyopathy clinical trials, including ATTR-CM: a systematic literature review
Journal of Comparative Effectiveness Research
Acute Hepatic Porphyria (AHP)
A randomized, placebo-controlled study of givosiran in patients with acute hepatic porphyrias (ENVISION): Final (36-month) analysis of the Taiwan Cohort
Journal of the Formosan Medical Association
Primary Hyperoxaluria Type 1 (PH1)
Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1: Results from a Phase III Clinical Trial
Kidney International Reports
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
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
