Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study
In the longest follow-up of givosiran-treated patients reported to date, the therapy maintained an acceptable safety profile and demonstrated sustained improvements in clinical outcomes over 4 years in patients with acute intermittent porphyria.
Authors
- Marianne T. Sweetser
- Bruce Wang (ORCID: https://orcid.org/0000-0003-3486-5494)
- David C. Rees (ORCID: https://orcid.org/0000-0003-3647-1050)
- Manisha Balwani (ORCID: https://orcid.org/0000-0001-8047-5011)
- Eliane Sardh (ORCID: https://orcid.org/0000-0002-2366-2213)
- Gang Jia (ORCID: https://orcid.org/0000-0003-1102-0683)
- Karl E. Anderson (ORCID: https://orcid.org/0000-0002-6499-8573)
Institutions
- Karolinska University Hospital (SE)
- Alnylam Pharmaceuticals (United States) (US)
- University of California, San Francisco (US)
- King's College Hospital (GB)
- The University of Texas Medical Branch at Galveston (US)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2024-10-03
- DOI
- https://doi.org/10.1186/s13023-024-03284-w
- Citations
- 8
- Primary Topic
- Porphyrin Metabolism and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 1.87
Funders
- Alnylam Pharmaceuticals
- Karolinska Institutet