Transthyretin Amyloidosis (ATTR)
Neurofilament light chain as a biomarker for hereditary ATTR amyloidosis
Amyloid
Acute Hepatic Porphyria (AHP)
Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study
Orphanet Journal of Rare Diseases
Acute Hepatic Porphyria (AHP)
High disease burden and healthcare resource usage in patients with acute porphyria—A population-based analysis
Liver International
Primary Hyperoxaluria Type 1 (PH1)
Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial
Frontiers in Pediatrics
Transthyretin Amyloidosis (ATTR)
Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy
New England Journal of Medicine
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
