Gene therapy is poised to break the stratosphere, providing many rare disease patients with an option out of their diseases. Whether innovations in CAR T-cell therapy or the rise of CRISPR, never before have patients had such a variety of therapeutic options becoming available. And the future is here. Just last December, the U.S. Food and Drug Administration (FDA) approved the first two cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older—one of which utilizes CRISPR technology. However, one glaring issue is blocking the path to success: who pays for the treatment? More information read here - https://bio.news/health/bio-2024-international-convention-gene-therapy-payment-models-bluebird-bio-vertex-pharmaceuticals/?fbclid=IwZXh0bgNhZW0CMTEAAR0Vn2LNtTHn4qcGwA7ffasyJQZyGJqzVut98f9J3OtWHTb9eCLK5PLr-Ik_aem_71qOwLShNmEfie4uslcTPA
TIF, its Board of Directors and its International Scientific Advisory Board proudly present the 4th Edition of the Federation’s most prestigious and renowned publication, the TIF Guidelines for The Management of Transfusion- Dependent Thalassaemia (TDT).
The latest TASCA newsletter is out now. Contents: Light Up Australia AGM Announcement Raffle Winners
World Hepatitis Day held on 28 July is an opportunity to step up national and international efforts on hepatitis. Although preventative measures such as donor and blood screening have increased blood safety and have virtually removed the risk of transfusion-transmitted infections in Australia, chronic hepatitis remains a concern in the genetic haemoglobin disorder community in Australia due to past infection and internationally due to still inadequate blood safety measures.
