The views of women with bleeding disorders: Results from the Cinderella study.

Introduction: Despite therapeutic advances in bleeding disorder treatment, over the past 20 years women with bleeding disorders have reported delayed diagnosis, impaired quality of life, dismissive attitudes from health professionals and inappropriate care.

Aim: To explore the lived experience of women with a diagnosed bleeding disorder and to improve understanding of their unmet needs.

Methods: Women haemophilia carriers (WHC), women with a diagnosed bleeding disorder (WBD) and women with immune thrombocytopenia (WITP) were invited to complete an online survey.

Parental perspectives on gene therapy for children with haemophilia: The Exigency study.

Introduction: Gene therapy is used in life-limiting conditions of childhood. While not a current therapeutic option for children with haemophilia, it may be considered in the future especially for those where access to treatment is limited.

Aim: To assess the attitudes and opinions of parents of children with haemophilia about gene therapy as a potential future treatment, by understanding their awareness about gene therapy and what they need to know now and in the future; gauging levels of interest in gene therapy for their children; and exploring perceived current motivations and barriers.