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
Acute Hepatic Porphyria (AHP)
Hyperhomocysteinemia in acute hepatic porphyria (AHP) and implications for treatment with givosiran
Expert Review of Gastroenterology & Hepatology
Acute Hepatic Porphyria (AHP)
Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study
Orphanet Journal of Rare Diseases
Transthyretin Amyloidosis (ATTR)
Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial
Amyloid
