Primary Hyperoxaluria Type 1 (PH1)
Quantification of oxalate by novel LC-MS/MS: assay development, validation and application in lumasiran clinical trials
Bioanalysis
Acute Hepatic Porphyria (AHP)
Pharmacokinetic-Pharmacodynamic Model of Urinary δ-Aminolevulinic Acid Reduction After Givosiran Treatment in Patients with Acute Hepatic Porphyria
CPT: Pharmacometrics & Systems Pharmacology
Primary Hyperoxaluria Type 1 (PH1)
Estimating health state utilities in primary hyperoxaluria type 1: a valuation study
Journal of Medical Economics
Acute Hepatic Porphyria (AHP)
Population Pharmacokinetic Analysis of the RNAi Therapeutic Givosiran in Patients with Acute Hepatic Porphyria
Clinical Pharmacokinetics
Transthyretin Amyloidosis (ATTR)
Effect of patisiran on stroke volume in hereditary transthyretin-mediated amyloidosis: insights from pressure–volume analysis of the APOLLO study
European Journal of Heart Failure
Transthyretin Amyloidosis (ATTR)
Impact of baseline polyneuropathy severity on patisiran treatment outcomes in the APOLLO trial
Amyloid
Acute Hepatic Porphyria (AHP)
EXPLORE B: A prospective, long-term natural history study of patients with acute hepatic porphyria with chronic symptoms
Journal of Inherited Metabolic Disease
Transthyretin Amyloidosis (ATTR)
Gene Silencing Therapeutics in Cardiology: A Review Article
International Journal of Cardiovascular Sciences
Primary Hyperoxaluria Type 1 (PH1)
Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial
Pediatric Nephrology
