Long-term lumasiran therapy final results from a Phase 2 open-label extension study in primary hyperoxaluria
These data represent the longest published follow-up of lumasiran-treated patients with PH1 (ages 6-43 years) to date. Long-term lumasiran treatment for PH1 had acceptable safety and led to sustained and substantial reduction of UOx with preservation of kidney function.
Authors
- Jaap W. Groothoff (ORCID: https://orcid.org/0000-0002-8467-3506)
- Jérôme Harambat (ORCID: https://orcid.org/0000-0003-2016-120X)
- John M. Gansner (ORCID: https://orcid.org/0000-0002-4030-3044)
- Wesley Nathan Hayes (ORCID: https://orcid.org/0000-0002-8091-6945)
- Anne‐Laure Sellier‐Leclerc (ORCID: https://orcid.org/0000-0001-9902-0850)
- Yaacov Frishberg
- Daniella Magen (ORCID: https://orcid.org/0000-0001-8825-5259)
- Martin Coenen
- Julien Hogan
- Richard E. Willey
- Sally‐Anne Hulton
Institutions
- Alnylam Pharmaceuticals (United States) (US)
- Inserm (FR)
- University Hospital Bonn (DE)
- Great Ormond Street Hospital (GB)
- Rambam Health Care Campus (IL)
- Shaare Zedek Medical Center (IL)
- Centre Hospitalier Universitaire de Bordeaux (FR)
- Hôpital Femme Mère Enfant (FR)
- Emma Kinderziekenhuis (NL)
- Hôpital Robert-Debré (FR)
- Amsterdam University Medical Centers (NL)
- Birmingham Women’s and Children’s NHS Foundation Trust (GB)
- University College London (GB)
- University of Amsterdam (NL)
Publication Details
- Journal
- Nephrology Dialysis Transplantation
- Published
- 2025-09-19
- DOI
- https://doi.org/10.1093/ndt/gfaf188
- Citations
- 1
- Primary Topic
- Kidney Stones and Urolithiasis Treatments
- Type
- article
- Field-Weighted Citation Impact
- 1.52
Funders
- Alnylam Pharmaceuticals