Acute Hepatic Porphyria (AHP)
Patient experience with acute hepatic porphyria before and after long-term givosiran treatment in a qualitative interview study
Molecular Genetics and Metabolism Reports
Transthyretin Amyloidosis (ATTR)
Worsening of Heart Failure in Outpatients With Transthyretin Amyloidosis and Cardiomyopathy in the APOLLO-B Trial
Journal of the American College of Cardiology
Transthyretin Amyloidosis (ATTR)
Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy
JAMA Neurology
Transthyretin Amyloidosis (ATTR)
The journey to diagnosis of wild-type transthyretin-mediated (ATTRwt) amyloidosis: a path with multisystem involvement
Orphanet Journal of Rare Diseases
Transthyretin Amyloidosis (ATTR)
Outpatient Worsening Heart Failure in Patients with Transthyretin Amyloidosis with Cardiomyopathy in the HELIOS-B Trial
Journal of the American College of Cardiology
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
