Safety and Efficacy of Long‐Term Treatment of Type 1 Plasminogen Deficient Patients With Intravenous Plasminogen Replacement Therapy
Long-term treatment with IV PLG concentrate is safe and effective for PLGD-1, demonstrating the potential for tailored dosing regimens. This study highlights the importance of individualised treatment and provides valuable insights into managing this ultra-rare disorder.
Authors
- Charles Nakar (ORCID: https://orcid.org/0009-0003-6301-0812)
- Jeremy Lorber (ORCID: https://orcid.org/0000-0002-4341-2721)
- Neelam Thukral
- Amy D. Shapiro (ORCID: https://orcid.org/0000-0003-2821-7159)
- Heather L. McDaniel (ORCID: https://orcid.org/0000-0001-8214-9551)
- Karen Thibaudeau (ORCID: https://orcid.org/0009-0008-6109-2953)
- Robert W. Decker
- Joseph M. Parker
Institutions
- Cedars-Sinai Medical Center (US)
- Indiana Hemophilia and Thrombosis Center (US)
- Vanderbilt University Medical Center (US)
Publication Details
- Journal
- Haemophilia
- Published
- 2025-03-18
- DOI
- https://doi.org/10.1111/hae.70019
- Citations
- 2
- Primary Topic
- Protease and Inhibitor Mechanisms
- Type
- article
- Field-Weighted Citation Impact
- 1.87