Work

Emerging therapies for hemophilia: controversies and unanswered questions

Year: 2018

Type: review

Abstract: Several new therapies for hemophilia have emerged in recent years. These strategies range from extended half-life factor replacement products and non-factor options with improved pharmacokinetic profiles to gene therapy aiming for phenotypic cure. While these products have the potential to change hemophilia care dramatically, several challenges and questions remain regarding broader applicability, long-term safety, and which option to pursue for each patient. Here, we review these emerging therapies with a focus on controversies and unanswered questions in each category.

Source: F1000Research

DOI: 10.12688/f1000research.12491.1

Authors: Valder R. Arruda, Bhavya S. Doshi, Benjamin J. Samelson-Jones

Institutions: Children's Hospital of Philadelphia, University of Pennsylvania

Language: English

Topic: Hemophilia Treatment and Research

Subfield: Hematology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $1,350