Primary Hyperoxaluria Type 1 (PH1)
Distinguishing characteristics of pediatric patients with primary hyperoxaluria type 1 in PEDSnet
Journal of Pediatric Urology
Acute Hepatic Porphyria (AHP)
Clinical features of acute attacks, chronic symptoms, and long-term complications among patients with acute hepatic porphyria in Japan: a real-world claims database study
Orphanet Journal of Rare Diseases
Transthyretin Amyloidosis (ATTR)
Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis
New England Journal of Medicine
Primary Hyperoxaluria Type 1 (PH1)
Qualitative assessment of the patient experience of primary hyperoxaluria type 1: an observational study
BMC Nephrology
Transthyretin Amyloidosis (ATTR)
Pharmacokinetics and Pharmacodynamics of Patisiran in Patients with hATTR Amyloidosis and with Polyneuropathy After Liver Transplantation
Clinical Pharmacokinetics
Transthyretin Amyloidosis (ATTR)
Impact of Vutrisiran on Quality of Life and Physical Function in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy
Neurology and Therapy
Cardiovascular (CV)
Zilebesiran, an RNA Interference Therapeutic Agent for Hypertension
New England Journal of Medicine
Acute Hepatic Porphyria (AHP)
Efficacy and safety of givosiran for acute hepatic porphyria: Final results of the randomized phase III ENVISION trial
Journal of Hepatology
Transthyretin Amyloidosis (ATTR)
Indirect treatment comparison (ITC) of the efficacy of vutrisiran and tafamidis for hereditary transthyretin-mediated amyloidosis with polyneuropathy
Expert Opinion on Pharmacotherapy
