Hemophilia gene therapy knowledge and perceptions: Results of an international survey.

ABSTRACT:

Background

Hemophilia gene therapy is a rapidly evolving therapeutic approach in which a number of programs are approaching clinical development completion.

Objective

The aim of this study was to evaluate knowledge and perceptions of a variety of health care practitioners and scientists about gene therapy for hemophilia.

Methods

This survey study was conducted February 1 to 18, 2019. Survey participants were members of the ISTH, European Hemophilia Consortium, European Hematology Association, or European Association for Hemophilia and Allied Disorders with valid email contacts. The online survey consisted of 36 questions covering demographic information, perceptions and knowledge of gene therapy for hemophilia, and educational preferences. Survey results were summariz

SUBMITTER: Peyvandi F 

PROVIDER: S-EPMC7292673 | biostudies-literature | 2020 May

REPOSITORIES: biostudies-literature