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Primary Hyperoxaluria Type 1 (PH1)
Primary Hyperoxaluria Type 1 (PH1) is a rare metabolic disorder caused by mutations in the alanine glyoxylate aminotransferase (AGXT) gene that result in a deficiency of liver-specific peroxisomal alanine glyoxylate aminotransferase (AGT) and consequent overproduction of oxalate by the liver. When the kidneys can no longer clear excess oxalate, calcium oxalate (CaOx) crystals deposit in other tissues, leading to systemic oxalosis.1
Resources
Leaflet
Therapies
OXLUMO® (lumasiran)
Explore clinical and educational resources related to the use of lumasiran in patients in all age groups with primary hyperoxaluria type 1 (PH1).
Congresses
Primary Hyperoxaluria Type 1 (PH1)
American Society of Nephrology (ASN) 2025
Primary Hyperoxaluria Type 1 (PH1)
European Society for Paediatric Nephrology (ESPN) 2025
Primary Hyperoxaluria Type 1 (PH1)
International Paediatric Transplant Association (IPTA) 2025
Publications
Primary Hyperoxaluria Type 1 (PH1)
Efficacy and Safety of Lumasiran for Advanced Primary Hyperoxaluria Type 1: 24-Month Follow-Up of the Phase 3 Illuminate-C Trial
American Journal of Kidney Diseases
References:
1. Fargue S, Acquaviva Bourdain C. Clin Kidney J. 2022;15(1):i4-i8.
Preparation Date: January 2026 Job Code: MB-UK-00039
