Patient experience with acute hepatic porphyria before and after long-term givosiran treatment in a qualitative interview study
Participants reported meaningful improvements in AHP symptoms, increased QOL, and reduced opioid use with long-term monthly givosiran treatment.
Authors
- Hetanshi Naik (ORCID: https://orcid.org/0000-0002-5894-7390)
- Stephen Lombardelli
- Michelle Brown (ORCID: https://orcid.org/0000-0001-9720-5616)
- Stephen Meninger
Institutions
- Alnylam Pharmaceuticals (United States) (US)
- Triangle (US)
- RTI Health Solutions (US)
- Icahn School of Medicine at Mount Sinai (US)
Publication Details
- Journal
- Molecular Genetics and Metabolism Reports
- Published
- 2024-12-20
- DOI
- https://doi.org/10.1016/j.ymgmr.2024.101174
- Citations
- 1
- Primary Topic
- Porphyrin Metabolism and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.23
Funders
- Alnylam Pharmaceuticals