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Patient Voices on Gene Therapy with Dr Priyanka Raheja

In these recordings, Dr Priyanka Raheja from the Royal Hospital in London, UK interviews a haemophilia patient who received his gene therapy infusion 7 years ago. Hear first hand experiences of the burden of haemophilia, the decision to start gene therapy, the lead up to infusion, the day of the infusion, and life after gene therapy.

Liver-related Aspects of Gene Therapy for Hemophilia: Need for Collaborations with Hepatologists

This paper by Wolfgang Miesbach and colleagues reviews liver-related gene therapy management in haemophilia and how this can be improved by close collaboration between haematologists and hepatologists.

Infrastructural Considerations of Implementing Gene Therapy for Hemophilia in the Nordic Context

In this paper Jan Astermark and colleagues set out to outline a function-based gene therapy working model, highlighting milestones in the handling, administration, and follow-up of gene therapy, allowing for the effective implementation of gene therapy in the Nordic region.

Patient Experience from South Africa

Listen to a firsthand account from a patient in South Africa sharing their journey with haemophilia gene therapy. Gain insights into their journey from growing up with haemophilia to life post-gene therapy.

About HaemDifferently.expert

Learn more about the free educational resources on haemophilia and gene therapy for healthcare professionals

Register for HaemDifferently.expert to discover a wide range of educational resources to help develop your expertise in haemophilia and gene therapy. Advance your knowledge with our publication summaries from key haemophilia publications over the last 70 years and educational videos on key topics in the field. Keep up to date with BioMarin’s congress brochures covering ISTH and EAHAD, listen to world-renowned experts discuss key topics including the patient gene therapy journey, Hub-and-Spoke model, and the multidisciplinary team, and download BioMarin’s patient- support resources to support facilitate your gene therapy conversations with patients.

Patient Journey

Free materials to support patients on the gene therapy journey

Our patient journey resources are designed to assist you in offering comprehensive support to patients navigating their haemophilia gene therapy journey. Dive into our resources for healthcare professionals to enhance your conversations with patients considering gene therapy. Among the many resources, you can find videos providing valuable insights into the practical aspects of gene therapy and discussions guides to facilitate meaningful conversations with patients around gene therapy.

Educational Content

Free slide summaries for a selection of publications on haemophilia and gene therapy.

The Steering Committee select the most relevant and up-to-date publications and slide summaries are developed for you to download. Explore summaries for key publications from the last 70 years, to develop your expertise in haemophilia and gene therapy.

Patient Voices on Gene Therapy

Hear firsthand experience from patients about their gene therapy journey

Watch our Patient Voices on Gene Therapy series with Dr Sylvia von Mackensen and Professor Hervé Chambost. Throughout this series, patients share their personal experience with gene therapy, including important factors in their decision-making process, the process of infusion and how gene therapy has impacted their lives.