Gene therapy and gene-modified cell therapies have a great potential for rare diseases to either help patients to cure their disease or improve their lives. Did you know that gene therapy will probably become a major treatment option for many rare diseases in the near future? Listen as expert hematologists delve into this topic and take hemophilia as a practical example. Join Prof. Cédric Hermans (Head of the Division of Haematology, the Hemostasis and Thrombosis Unit and the Hemophilia Center of the Saint-Luc University Hospital in Brussels, Belgium) and Prof. Miguel Escobar (Professor of Medicine and Pediatrics, University of Texas Health Science Center at Houston-McGovern Medical School and University of Texas M.D. Anderson Cancer Center Medical Director, Gulf States Hemophilia & Thrombophilia Center Houston, USA) as they engage in a conversation about the potential of gene and gene-modified cell therapies in rare diseases, and how early gene therapies have been implemented as a treatment approach for rare diseases, such as leber congenital amaurosis, spinal muscular atrophy, beta-thalassemia, adrenoleukodystrophy, and hemophilia. The experts explore hemophilia as an example of how gene therapy has become a reality within rare diseases. They discuss why we need gene therapy for rare disease, what gene therapy and gene modified cell therapy is, and how this has become a reality in hemophilia, and look to the future of gene therapy for rare diseases. Finally they reflect on the importance of a multidisciplinary approach. - Access information on the programme, the clinical takeaways, the flashcard and the transcript here https://cor2ed.com/hemostasis-connect/programmes/gene-therapy-rare-diseases/ Find out about the experts Prof Cedric Hermans and Prof. Miguel Escobar. The medical experts in this podcast are expressing their own views and not those of COR2ED, supporters, or their institution. This podcast is supported by an Independent Medical Education Grant from The American Society of Gene + Cell Therapy and Pfizer.

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